Decision

Orphan register

Updated 18 December 2024

Orphan registered products are listed in alphabetical order by trade name.

The list may be searched by pressing “CTRL+F” and entering a key word or phrase.

Where you are instructed to see a numbered section for more information, you can do so in the Summary of Product Characteristics (SmPC) for each product.

ABECMA

Active substance: idecabtagene vicleucel    

Orphan condition: Multiple myeloma

Authorised orphan indication: Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy

Orphan market exclusivity expiry date: 23 June 2032

GB Orphan Designation Number: PLGB 50412/0023/OD1

ADAKVEO

Active substance: crizanlizumab

Orphan condition: Sickle cell disease

Authorised orphan indication:

Adakveo is indicated for the prevention of recurrent vaso-occlusive crises (VOCs) in sickle cell disease patients aged 16 years and older. It can be given as an add on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.

Orphan market exclusivity date: 29 October 2030

GB Orphan Designation Number: PLGB 00101/1191/OD1

ADCETRIS

Active substance: brentuximab vedotin

Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E

Orphan condition: Cutaneous T cell lymphoma

Authorised orphan indication:

ADCETRIS is indicated for the treatment of adult patients with CD30+ cutaneous T cell lymphoma (CTCL) after at least 1 prior systemic therapy

Orphan market exclusivity expiry date: 19 December 2027

GB Orphan designation number: PLGB 16189/0093/OD3

AGAMREE

Active substance: VAMOROLONE  

Orphan condition: Duchenne muscular dystrophy

Authorised orphan indication: Vamorolone in the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

Orphan market exclusivity date: 11/01/2034

GB Orphan Designation Number: PLGB 25835/0005/OD1

Alofisel

Active substance: Expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue

Orphan condition: Anal fistula

Authorised orphan indication:

Alofisel is indicated for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease, when fistulas have shown an inadequate response to at least one conventional or biologic therapy. Alofisel should be used after conditioning of fistulas.

Orphan market exclusivity expiry date: 27 March 2028

GB Orphan designation number: PLGB 16189/0094/OD1

ALPROLIX

Active substance: eftrenonacog alfa

Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1

Orphan condition: Haemophilia B (congenital factor IX deficiency)

Authorised orphan indication:

Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).

ALPROLIX can be used for all age groups.

Orphan market exclusivity expiry date: 13 May 2026

GB Orphan designation number: PLGB 30941/0003 – 0007/OD1

AMGLIDIA

Active substance: Glibenclamide

Orphan condition: Neonatal diabetes

Authorised orphan indication:

AMGLIDIA is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children.

Sulphonylureas like AMGLIDIA have been shown to be effective in patients with mutations in the genes coding for the β-cell ATP-sensitive potassium channel and chromosome 6q24-related transient neonatal diabetes mellitus.

Orphan market exclusivity expiry date: 28 May 2028

GB Orphan designation number: PLGB 50687/0001 – 0004/OD1

AMVUTTRA

Active substance: Vutrisiran Sodium

Orphan condition: Transthyretin-mediated amyloidosis

Authorised orphan indication:

Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.

Orphan market exclusivity expiry date: 16 September 2032

GB Orphan designation number: PLGB 50597/0006/OD1

ARIKAYCE liposomal

Active substance: Amikacin sulfate

Orphan condition: Non-tuberculous mycobacterial lung disease

Authorised orphan indication:

ARIKAYCE liposomal is indicated for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis.

Orphan market exclusivity expiry date: 28 October 2030

Orphan designation number: PLGB 47434/0001/OD1

Artesunate Amivas

Active substance: Artesunate

Orphan condition: Malaria

Authorised orphan indication: Artesunate Amivas is indicated for the initial treatment of severe malaria in adults and children

Orphan market exclusivity expiry date: 27 March 2032

GB Orphan Designation Number: PLGB 55184/0002 /OD1

ASPAVELI

Active substance: pegcetacoplan

Orphan condition: Paroxysmal nocturnal haemoglobinuria

Authorised orphan indication: ASPAVELI is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.

Orphan market exclusivity expiry date: 24 February 2032

GB Orphan Designation Number: PLGB 30941/0022/OD1

AYVAKYT

Active substance: avapritinib

(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine

Orphan condition: Gastrointestinal stromal tumours

Authorised orphan indication:

AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation

Orphan market exclusivity expiry date: 25 September 2030

Orphan designation number: PLGB 52115/0001 – 0003/0D1

AYVAKYT

Active substance: avapritinib

(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine

Orphan condition: Mastocytosis

Authorised orphan indication: AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL).

Orphan market exclusivity expiry date: 10/09/2034

Orphan designation number: PLGB 52115/0001 – 0002/OD2, PLGB 52115/0006 – 0007/OD2

Besponsa

Active substance: Inotuzumab ozogamicin

Orphan condition: B-cell acute lymphoblastic leukaemia

Authorised orphan indication:

BESPONSA is indicated as monotherapy for the treatment of adults with relapsed or refractory CD22-positive B cell precursor acute lymphoblastic leukaemia (ALL). Adult patients with Philadelphia chromosome positive (Ph+) relapsed or refractory B cell precursor ALL should have failed treatment with at least 1 tyrosine kinase inhibitor (TKI).

Orphan market exclusivity expiry date: 3 July 2027

GB Orphan designation number: PLGB 00057/1546/OD1

BLINCYTO

Active substance: Blinatumomab

Orphan condition: Acute lymphoblastic leukaemia

Authorised orphan indication:

BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.

BLINCYTO is indicated as monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation.

BLINCYTO is indicated for the treatment of adult patients with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in the consolidation phase.

BLINCYTO is indicated for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in first relapse in the consolidation phase.

Orphan market exclusivity expiry date: 25 November 2027

GB Orphan designation number: PLGB 13832/0018/OD1

Brineura

Active substance: cerliponase alfa

Recombinant human tripeptidyl-peptidase 1

Orphan condition: Neuronal ceroid lipofuscinosis type 2 disease

Authorised orphan indication:

Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

Orphan market exclusivity expiry date: 1 June 2027

GB Orphan designation number: PLGB 45814/0001/OD1

Bylvay

Orphan condition: Progressive familial intrahepatic cholestasis

Authorised orphan indication: Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.

Orphan market exclusivity expiry date: 04 August 2031

GB Orphan designation number: PLGB 36216/0001-0004/OD1

Cablivi

Active substance: caplacizumab

Nanobody directed towards the human A1 domain of von Willebrand factor

Orphan condition: Thrombotic thrombocytopenic purpura

Authorised orphan indication:

Cablivi is indicated for the treatment of adults and adolescents of 12 years of age and older weighing at least 40 kg experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.

Orphan market exclusivity expiry date: 4 September 2030

GB Orphan designation number: PLGB 04425/0888/OD1

Carvykti

Active substance: Ciltacabtagene autoleucel

Orphan condition: Multiple myeloma

Authorised orphan indication:

Treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.

Orphan market exclusivity expiry date: 4 January 2033

GB Orphan Designation Number: PLGB 00242/0745/OD1

Casgevy

Active substance: autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene

Orphan condition: β thalassaemia intermedia and major

Authorised orphan indication: treatment of transfusion-dependent β-thalassemia in patients 12 years of age and older for whom a human leukocyte antigen-matched related haematopoietic stem cell donor is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available.

Orphan market exclusivity expiry date: 15 November 2033

GB Orphan designation number: PLGB 22352/0019/OD1

Casgevy

Active substance: autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene

Orphan condition: sickle cell disease

Authorised orphan indication: treatment of sickle cell disease in patients 12 years of age and older  with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom a haematopoeitic stem cell transplantation is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available.

Orphan market exclusivity expiry date: 15 November 2033

GB Orphan designation number: PLGB 22352/0019/OD2

Cerdelga

Active substance: eliglustat

Orphan condition: Gaucher Disease

(1R, 2R)-Octanoic acid [2-(2’,3’-dihydro-benzo [1,4] dioxin-6’-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt

Authorised orphan indication:

Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).

Orphan market exclusivity expiry date: 21 January 2025

GB Orphan designation number: PLGB 04425/0763/OD1

Chenodeoxycholic acid Leadiant

Active substance: Chenodeoxycholic acid

Orphan condition: Inborn errors of primary bile acid synthesis

Authorised orphan indication:

Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults

Orphan market exclusivity expiry date: 12 April 2027

GB Orphan designation number: PLGB 44975/0001/OD1

Coagadex

Active substance: Human coagulation factor X

Orphan condition: Hereditary factor X deficiency

Authorised orphan indication:

Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency.

Coagadex is indicated in all age groups.

Orphan market exclusivity expiry date: 18 March 2028

GB Orphan designation number: PLGB 08801/0059 – 0060/OD1

Columvi 2.5 mg and 10mg concentrate for solution for infusion  

Active substance: GLOFITAMAB

Orphan condition: Diffuse large B-cell lymphoma

Authorised orphan indication: Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL), after two or more lines of systemic therapy.

Orphan market exclusivity expiry date: 16 October 2033

GB Orphan designation number: PLGB 00031/0931-0932/OD1

Cresemba

Active substance: isavuconazole

Isavuconazonium sulfate

Orphan condition: Mucormycosis

Authorised orphan indication:

Treatment of mucormycosis in patients for whom amphotericin B is inappropriate

Consideration should be given to official guidance on the appropriate use of antifungal agents.

Orphan market exclusivity expiry date: 19 Octoeber 2025

GB Orphan designation number: PLGB 32205/0005 – 0006/OD1

Cresemba

Active substance: isavuconazole

Isavuconazonium sulfate

Orphan condition: Invasive aspergillosis

Authorised orphan indication:

Treatment of invasive aspergillosis

Consideration should be given to official guidance on the appropriate use of antifungal agents.

Orphan market exclusivity expiry date: 19 October 2025

GB Orphan designation number: PLGB 32205/0005 – 0006/OD2

CRYSVITA

Active substance: Burosumab

Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23

Orphan condition: X-linked hypophosphataemia

Authorised orphan indication:

CRYSVITA is indicated for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons

Orphan market exclusivity expiry date: 21 February 2028

GB Orphan designation number: PLGB 50262/0001/OD1

Cystadrops

Active substance: mercaptamine

Cysteamine hydrochloride

Orphan condition: Cystinosis

Authorised orphan indication:

Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis.

Orphan market exclusivity expiry date: 23 January 2027

GB Orphan designation number: PLGB 15266/0021/OD1

Darzalex

Active substance: Daratumumab

Orphan condition: Plasma cell myeloma

Authorised orphan indication:

DARZALEX is indicated:

• in combination with lenalidomide and dexamethasone or with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant
• in combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant.
• in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant
• in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy
• in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received one prior therapy containing a proteasome inhibitor and lenalidomide and were lenalidomide-refractory, or who have received at least two prior therapies that included lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or after the last therapy (see section 5.1)
• as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy

Orphan market exclusivity expiry date: 24 May 2026

GB Orphan designation number: PLGB 00242/0676 – 0677/OD1

Darzalex

Orphan condition: Systemic light chain (AL) amyloidosis

Authorised orphan indication: Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis

Orphan market exclusivity expiry date: 11 October 2031

GB Orphan designation number: PLGB 00242/0677/OD2

Daurismo

Active substance: glasdegib maleate

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

Daurismo is indicated, in combination with low-dose cytarabine, for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy.

Orphan market exclusivity expiry date: 29 June 2030

GB Orphan designation number: PLGB 00057/1687 – 1688/OD1

Dovprela

Active substance: pretomanid

(S)-2-nitro-6-(4-(trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b][1,3]oxazine

Orphan condition: Tuberculosis

Authorised orphan indication:

Dovprela is indicated in combination with bedaquiline and linezolid, in adults, for the treatment of pulmonary extensively drug resistant (XDR), or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB).

Orphan market exclusivity expiry date: 4 August 2030

GB Orphan designation number: PLGB 46302/0234/OD1

Ebvallo 2.8 x 10^7 - 7.3 x 10^7 cells/mL dispersion for injection

Active substance: TABELECLEUCEL

Orphan condition: Post transplant Epstein-Barr virus associated lymphoproliferative disorder

Authorised orphan indication: treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate’.

Orphan market exclusivity expiry date: 22 May 2033

GB Orphan designation number: PLGB 52297/0001/OD1

ELZONRIS

Active substance: TAGRAXOFUSP

Orphan condition: Blastic plasmacytoid dendritic cell neoplasia

Authorised orphan indication: Monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm

Orphan market exclusivity expiry date: 14 October 2031

GB Orphan designation number: PLGB 53425/0001/OD1

Epidyolex

Active substance: cannabidiol

Orphan condition: Lennox-Gastaut syndrome

Authorised orphan indication:

Epidyolex is indicated for use as adjunctive therapy of seizures associated with Lennox Gastaut syndrome (LGS), in conjunction with clobazam, for patients 2 years of age and older.

Orphan market exclusivity expiry date: 23 September 2029

GB Orphan designation number: PLGB 36772/0001/OD1

Enspryng

Active substance: Satralizumab

Orphan condition: Neuromyelitis optica spectrum disorders (NMOSD)

Authorised orphan indication: Enspryng is indicated as a monotherapy or in combination with immunosuppressive therapy (IST) for the treatment of neuromyelitis optica spectrum disorders (NMOSD) in adult and adolescent patients from 12 years of age who are anti-aquaporin-4 IgG (AQP4-IgG) seropositive.

Orphan market exclusivity expiry date: 21/11/2033

GB Orphan Designation Number: PLGB 00031/0916 /OD1

Epidyolex

Active substance: cannabidiol

Orphan condition: Dravet syndrome

Authorised orphan indication:

Epidyolex is indicated for use as adjunctive therapy of seizures associated with Dravet syndrome (DS), in conjunction with clobazam, for patients 2 years of age and older.

Orphan market exclusivity expiry date: 23 September 2029

GB Orphan designation number: PLGB 36772/0001/OD2

Epidyolex

Orphan condition: Tuberous sclerosis complex

Authorised orphan indication: Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older.

Orphan market exclusivity expiry date: 05 August 2031

GB Orphan designation number: PLGB 36772/0001/OD3

FABHALTA

Active substance: IPTACOPAN HYDROCHLORIDE MONOHYDRATE  

Orphan condition: Paroxysmal Nocturnal Haemoglobinuria (PNH)

Authorised orphan indication: Monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia

Orphan market exclusivity expiry date: 02/08/2034

GB Orphan Designation Number: PLGB  00101/1231/OD1

Farydak

Active substance: panobinostat

Orphan condition: Treatment of multiple myeloma

Authorised orphan indication:

Farydak, in combination with bortezomib and dexamethasone, is indicated for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent.

Orphan market exclusivity expiry date: 1 September 2025

GB Orphan designation number: PLGB 53958/0007 – 0009/OD1

Filspari

Active substance: Sparsentan

Orphan condition: Primary IGA Nephropathy

Authorised orphan indication: treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1.0 g/day (or urine protein-to-creatinine ratio ≥ 0.75 g/g)

Orphan market exclusivity expiry date: 06/11/2034

GB Orphan Designation Number: PLGB 15240/0005/OD1 & PLGB 15240/0006/OD1

FILSUVEZ

Active substance: BETULAE CORTEX DRY EXTRACT

Orphan condition: Epidermolysis bullosa

Authorised orphan indication: Treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients 6 months and older

Orphan market exclusivity date: : 11 August 2032

GB Orphan Designation Number: PLGB 50688/0011/OD1

FINLEE

Active substance: DABRAFENIB MESYLATE  

Orphan condition: Glioma

Authorised orphan indication: 

Low-grade glioma

Finlee in combination with trametinib is indicated for the treatment of

paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.

High-grade glioma

Finlee in combination with trametinib is indicated for the treatment of

paediatric patients aged 1 year and older with high-grade glioma

(HGG) with a BRAF V600E mutation who have received at least one

prior radiation and/or chemotherapy treatment.

Orphan market exclusivity expiry date: 20/03/2036

GB Orphan Designation Number: PLGB  00101/1228/OD1

Fintepla

Active substance: fenfluramine

Orphan condition: Dravet syndrome

Authorised orphan indication:

Fintepla is indicated for the treatment of seizures associated with Dravet syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older.

Orphan market exclusivity expiry date: 21 December 2030

GB Orphan designation number: PLGB 45831/0001/OD1

Fintepla

Active substance: fenfluramine hydrochloride

Orphan condition: Lennox-Gastaut syndrome

Authorised orphan indication:

Treatment of seizures associated with Lennox- Gastaut syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older.

Orphan market exclusivity expiry date: 5 July 2033

GB Orphan designation number: PLGB 00039/0804 – 0010OD2

Galafold

Active substance: migalastat

1-deoxygalactonojirimycin hydrochloride

Orphan condition: Fabry disease

Authorised orphan indication:

Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1).

Orphan market exclusivity expiry date: 31 May 2026

GB Orphan designation number: PLGB 25823/0002/OD1

Gazyvaro

Active substance: obinutuzumab

Orphan condition: Follicular lymphoma (FL)

Authorised orphan indication:

Gazyvaro in combination with chemotherapy, followed by Gazyvaro maintenance therapy in patients achieving a response, is indicated for the treatment of patients with previously untreated advanced FL (see section 5.1)

Gazyvaro in combination with bendamustine followed by Gazyvaro maintenance is indicated for the treatment of patients with FL who did not respond or who progressed during or up to 6 months after treatment with rituximab or a rituximab-containing regimen.

Orphan market exclusivity expiry date: 15 June 2026

GB Orphan designation number: PLGB 00031/0856/OD2

Givlaari

Active substance: givosiran

Orphan condition: Acute hepatic porphyria

Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA covalently linked to a ligand containing three N-acetylgalactosamine residues

Authorised orphan indication:

Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older

Orphan market exclusivity expiry date: 4 March 2030

GB Orphan designation number: PLGB 43942/0001/OD1

Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion

Active substance: ETRANACOGENE DEZAPARVOVEC

Orphan condition: Haemophilia B (Factor IX)

Authorised orphan indication: treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.

Orphan market exclusivity expiry date: 22 March 2033

GB Orphan designation number: PLGB 15036/0160/OD1

Hetlioz

Active substance: tasimelteon

Orphan condition: Non-24-hour sleep-wake disorders in blind people with no light perception

Authorised orphan indication:

HETLIOZ is indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (Non-24) in totally blind adults.

Orphan market exclusivity expiry date: 7 July 2025

GB Orphan designation number: PLGB 43460/0001/OD1

Holoclar

Active substance:

Ex-vivo expanded autologous human corneal epithelium containing stem cells

Orphan condition: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns

Authorised orphan indication:

Treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy.

Orphan market exclusivity expiry date: 19 February 2025

GB Orphan designation number: PLGB 53298/0001/OD1

Hyftor

Active substance: SIROLIMUS

Orphan condition: Tuberous sclerosis

Authorised orphan indication: Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older

Orphan market exclusivity expiry date: 1 September 2033

GB Orphan designation number: PLGB 57572/0001/OD1

Idefirix

Active substance: imlifidase

Recombinant IgG degrading enzyme of Streptococcus pyogenes

Orphan condition: Graft rejection following solid organ transplantation

Authorised orphan indication:

Idefirix is indicated for desensitisation treatment of highly sensitised adult kidney transplant patients with positive crossmatch against an available deceased donor. The use of Idefirix should be reserved for patients unlikely to be transplanted under the available kidney allocation system including prioritisation programmes for highly sensitised patients.

Orphan market exclusivity expiry date: 1 September 2030

GB Orphan designation number: PLGB 46323/0002/OD1

IDELVION

Active substance: albutrepenonacog alfa

Recombinant fusion protein linking human coagulation factor IX with human albumin

Orphan condition: Haemophilia B

Authorised orphan indication:

Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency).

IDELVION can be used for all age groups.

Orphan market exclusivity expiry date: 13 May 2026

GB Orphan designation number: PLGB 00231/0337 – 0340/OD1

Imcivree

Active substance: setmelanotide

Orphan condition: genetic obesity disorders caused by defects in melanocortin 4 receptor (MC4R) pathway, including POMC and LEPR deficiency obesity

Authorised orphan indication:

The treatment of obesity and the control of hunger associated with genetically confirmed loss of function biallelic pro-opiomelanocortin (POMC), including PCSK1 deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above

Orphan market exclusivity expiry date: 14 September 2031

GB Orphan Designation Number: PLGB 55587/0001/OD1

Imcivree

Active substance: setmelanotide

Orphan condition: Bardet-Biedl syndrome (BBS)

Authorised orphan indication:

The treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) in adults and children 2 years of age and above

Orphan market exclusivity date: 17 November 2032

GB Orphan Designation Number: PLGB 55587/0001/OD2

Inrebic

Active substance: fedratinib dihydrochloride monohydrate

Orphan condition: Myelofibrosis

Authorised orphan indication:

Inrebic is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.

Orphan market exclusivity expiry date: 16 April 2031

GB Orphan designation number: PLGB 50412/0022/OD1

Isturisa

Active substance: osilodrostat

Orphan condition: Cushing’s syndrome

Authorised orphan indication:

Isturisa is indicated for the treatment of endogenous Cushing’s syndrome in adults.

Orphan market exclusivity expiry date: 13 Jan 2030

GB Orphan designation number: PLGB 15266/0029 – 0031/OD1

IQIRVO

Active substance: Elafibranor

Orphan condition: Primary Biliary Cholangitis

Authorised orphan indication:  Iqirvo is indicated for the treatment of primary biliary cholangitis.

Orphan market exclusivity expiry date: 1 October 2034

GB Orphan Designation Number: PLGB 34926/0029/OD1

Joenja

Joenja 70 mg film-coated tablets

Active substance: LENIOLISIB

Orphan condition: Activated phosphoinositide 3 kinase delta (P13Kδ) syndrome (APDS)

Authorised orphan indication: Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta (P13Kδ) syndrome (APDS) in adult and paediatric patients 12 years of age and older.

Orphan market exclusivity expiry date: 25/09/2034

GB Orphan designation number: PLGB 33010/0001/OD1

Jorveza

Active substance: budesonide

Orphan condition: Eosinophilic oesophagitis

Authorised orphan indication:

Jorveza is indicated for the treatment of eosinophilic esophagitis (EoE) in adults (older than 18 years of age).

Orphan market exclusivity expiry date: 10 Jan 2028

GB Orphan designation number: PLGB 08637/0030-0032/OD1

Kaftrio

Active substance: ivacaftor, tezacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide (elexacaftor)

Orphan condition: Cystic fibrosis

Authorised orphan indication:

Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see section 5.1).

Orphan market exclusivity expiry date: 21 August 2030

GB Orphan designation number: PLGB 22352/0012/OD1

GB Orphan Designation number: PLGB 22352/0017/OD1

Kanuma

Active substance: sebelipase alfa

Recombinant human lysosomal acid lipase

Orphan condition: Lysosomal acid lipase deficiency

Authorised orphan indication:

Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) Deficiency.

Orphan market exclusivity expiry date: 1 September 2025

GB Orphan designation number: PLGB 31775/0002/OD1

Kinpeygo

Active substance: Budesonide

Orphan condition: Primary immunoglobulin A (IgA) nephropathy

Authorised orphan indication:

Treatment of primary immunoglobulin A (IgA) nephropathy (IgAN) in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram

Orphan market exclusivity expiry date: 1 February 2033

GB Orphan designation number: PLGB 49329/0001/OD1

KIMMTRAK

Active substance: tebentafusp

Orphan condition: Uveal melanoma

Authorised orphan indication:

KIMMTRAK is indicated as monotherapy for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma.

Orphan market exclusivity expiry date: 6 June 2032

GB Orphan designation number: PLGB 36781/0001/OD1

Koselugo

Active substance: selumetinib hydrogen sulfate

Orphan condition: Neurofibromatosis Type 1

Authorised orphan indication: Treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above.

Orphan market exclusivity expiry date: 8 August 2031

GB Orphan Designation Number: PLGB 17901/0356 - 0357/OD1

Kymriah

Active substance: tisagenlecleucel

Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19

Orphan condition: B-lymphoblastic leukaemia

Authorised orphan indication:

Kymriah is indicated for the treatment of paediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post transplant or in second or later relapse.

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 00101/1101/OD1

Kymriah

Active substance: tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19

Orphan condition: Diffuse large B-cell lymphoma

Authorised orphan indication:

Kymriah is indicated for the treatment of:

• Adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy.

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 00101/1101/OD2

KYMRIAH

Active substance: Tisagenlecleucel

Orphan condition: Follicular lymphoma

Authorised orphan indication: treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy

Orphan market exclusivity expiry date: 9 August 2032

GB Orphan Designation Number: PLGB 00101/1101/OD3

Kyprolis

Active substance: carfilzomib

Orphan condition: Multiple myeloma

Authorised orphan indication:

Kyprolis in combination with daratumumab and dexamethasone, with lenalidomide and dexamethasone, or with dexamethasone alone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.

Orphan market exclusivity expiry date: 23 November 2025

GB Orphan designation number: PLGB 13832/0023 – 0025/OD1

Lamzede

Active substance: velmanase alfa

Recombinant human α-Mannosidase

Orphan condition: α-Mannosidosis

Authorised orphan indication:

Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha mannosidosis. See sections 4.4 and 5.1.

Orphan market exclusivity expiry date: 27 March 2028

GB Orphan designation number: PLGB 08829/0188/OD1

Ledaga

Active substance: chlormethine

Orphan condition: Cutaneous T-cell lymphoma

Authorised orphan indication:

Ledaga is indicated for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF type CTCL) in adult patients

Orphan market exclusivity expiry date: 7 March 2027 GB Orphan designation number: PLGB 15720/0005/OD1

Libmeldy

Active substance: Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene

Orphan condition: Metachromatic leukodystrophy

Authorised orphan indication:

Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:

• in children with late infantile or early juvenile forms, without clinical manifestations of the disease,
• in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline

Orphan market exclusivity expiry date: 18 December 2030

GB Orphan designation number: PLGB 49055/0002/OD1

LIVMARLI

Active substance: maralixibat chloride

Orphan condition: Cholestatic pruritus

Authorised orphan indication: Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older

Orphan market exclusivity expiry date: 10 February 2035

GB Orphan designation number: PLGB 56642/0001/OD1

LIVTENCITY

Active substance: maribavir

Orphan condition: Cytomegalovirus (CMV) infection in patients with impaired cell-mediated immunity

Authorised orphan indication:

Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents.

Orphan market exclusivity expiry date: 11 November 2032

GB Orphan Designation Number: PLGB 16189/0127/OD1

LIVTENCITY

Active substance:  maribavir

Orphan condition: Cytomegalovirus (CMV) disease in patients with impaired cell-mediated immunity

Authorised orphan indication:

Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents

Orphan market exclusivity expiry date: 11 November 2032

GB Orphan Designation Number: PLGB 16189/0127/OD2

Loargys 5 mg/ml solution for injection/infusion  

Active substance: PEGZILARGINASE  

Orphan condition: Arginase 1 deficiency

Authorised orphan indication:

Treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.

Orphan market exclusivity expiry date: 20 December 2033

GB Orphan designation number: PLGB 53487/0007/OD1

Lonapegsomatropin Ascendis Pharma

Active substance: lonapegsomatropin

Orphan condition: Growth hormone deficiency

Authorised orphan indication:

Growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD])

Orphan market exclusivity expiry date: 17 October 2032

GB Orphan designation number: PLGB 47497/0001-009/OD1

LUNSUMIO

Active substance: mosunetuzumab

Orphan condition: Follicular lymphoma

Authorised orphan indication:

Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least two prior systemic therapies

Orphan market exclusivity expiry date:  4 October 2032

GB Orphan designation number: PLGB 00031/0933-0934/OD1

Lutathera

Active substance: lutetium (177Lu) oxodotreotide

Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl

Orphan condition: Gastro-entero-pancreatic neuroendocrine tumours

Authorised orphan indication:

Lutathera is indicated for the treatment of unresectable or metastatic, progressive, well differentiated (G1 and G2), somatostatin receptor positive gastroenteropancreatic neuroendocrine tumours (GEP NETs) in adults.

Orphan market exclusivity expiry date: 28 September 2027

GB Orphan designation number: PLGB 35145/0003/OD1

Luxturna

Active substance: voretigene neparvovec

Adenovirus associated viral vector serotype 2 containing the human RPE65 gene

Orphan condition: Inherited retinal dystrophies (initially named Leber’s congenital amaurosis)

Authorised orphan indication:

Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

Orphan market exclusivity expiry date: 5 December 2028

GB Orphan designation number: PLGB 00101/1104/OD1

Luxturna

Active substance: voretigene neparvovec

Adenovirus associated viral vector serotype 2 containing the human RPE65 gene

Orphan condition: Inherited retinal dystrophies (initially named retinitis pigmentosa)

Authorised orphan indication:

Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

Orphan market exclusivity expiry date: 5 December 2028

GB Orphan designation number: PLGB 00101/1104/OD2

Mepsevil

Active substance: vestronidase alfa

Recombinant human beta-glucuronidase

Orphan condition: Mucopolysaccharidosis type VII (Sly syndrome)

Authorised orphan indication:

Mepsevil is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 474793/0002/OD1

Minjuvi

Active substance: tafasitamab

Orphan condition: Relapsed or refractory diffuse large B-cell lymphoma

Authorised orphan indication:

Minjuvi is indicated in combination with lenalidomide followed by Minjuvi monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT).

Orphan market exclusivity expiry date: 7 October 2031

GB Orphan Designation Number: PLGB 42338/0016/OD1

Myalepta

Active substance: metreleptin

Orphan condition: Familial Partial Lipodystrophy

Authorised orphan indication:

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:

• with confirmed familial partial LD in adults and children 12 years of age and above for whom standard treatments
• have failed to achieve adequate metabolic control.

Orphan market exclusivity expiry date: 1 August 2028

GB Orphan designation number: PLGB 50688/0008-0010/OD1

Myalepta

Active substance: metreleptin

Orphan condition: Barraquer-Simons syndrome

Authorised orphan indication:

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:

with acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.

Orphan market exclusivity expiry date: 1 August 2028

GB Orphan designation number: PLGB 50688/0008-0010/OD2

Myalepta

Active substance: metreleptin

Orphan condition: Lawrence syndrome

Authorised orphan indication:

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above

Orphan market exclusivity expiry date: 1 August 2028

GB Orphan designation number: PLGB 50688/0008-0010/OD3

Myalepta

Active substance: metreleptin

Orphan condition: Berardinelli-Seip syndrome

Authorised orphan indication:

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome)

Orphan market exclusivity expiry date: 1 August 2028

GB Orphan designation number: PLGB 50688/0008-0010/OD4

Mylotarg

Active substance: gemtuzumab ozogamicin

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

MYLOTARG is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33 positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL).

Orphan market exclusivity expiry date: 23 April 2028

GB Orphan designation number: PLGB 00057/1591/OD1

Namuscla

Active substance: mexiletine hydrochloride

Orphan condition: Myotonic disorders

Authorised orphan indication:

Namuscla is indicated for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders

Orphan market exclusivity expiry date: 20 December 2028

GB Orphan designation number: PLGB 35507/0200/OD1

Natpar

Active substance: parathyroid hormone

Recombinant human parathyroid hormone

Orphan condition: Hypoparathyroidism

Authorised orphan indication:

Natpar is indicated as adjunctive treatment of adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone.

Orphan market exclusivity expiry date: 26 April 2027

GB Orphan designation number: PLGB 16189/0067 – 0070/OD1

Nexviadyme 100 mg powder for concentrate for solution for infusion

Active substance: AVALGLUCOSIDASE ALFA

Orphan condition: Glycogen storage disease type II (Pompe’s disease)

Authorised orphan indication: Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency)

Orphan market exclusivity expiry date: 3 May 2033

GB Orphan designation number: PLGB 04425/0893/OD1

Ngenla

Active substance: somatrogon

Orphan condition:

Growth hormone deficiency

Authorised orphan indication:

Ngenla is indicated for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone.

Orphan market exclusivity expiry date: 25 March 2032

GB Orphan Designation Number: PLGB 00057/1712-1713/OD1

Ninlaro

Active substance: ixazomib

2,2’-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid

Orphan condition:

Multiple myeloma

Authorised orphan indication:

NINLARO in combination with lenalidomide and dexamethasone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.

Orphan market exclusivity expiry date: 23 November 2026

GB Orphan designation number: PLGB 16189/0109 – 0111/OD1

Obiltoxaximab SFL

Active substance: obiltoxaximab

Orphan condition: Anthrax

Authorised orphan indication:

Obiltoxaximab SFL is indicated in combination with appropriate antibacterial drugs in all age groups for treatment of inhalational anthrax due to Bacillus anthracis (see section 5.1).

Obiltoxaximab SFL is indicated in all age groups for post-exposure prophylaxis of inhalational anthrax when alternative therapies are not appropriate or are not available (see section 5.1).

Orphan market exclusivity expiry date: 19 November 2030

GB Orphan designation number: PLGB 54280/0001/OD1

Ocaliva

Active substance: obeticholic acid

6alpha-ethyl-chenodeoxycholic acid

Orphan condition: Primary biliary cirrhosis

Orphan condition: Primary biliary cirrhosis

Authorised orphan indication:

Ocaliva is indicated for the treatment of primary biliary cholangitis (PCB) (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

Orphan market exclusivity expiry date: 15 December 2026

GB Orphan designation number: PLGB 48025/0002 – 0003/OD1

Onivyde pegylated liposomal

Active substance: irinotecan

Nanoliposomal irinotecan

Orphan condition: Pancreatic cancer

Authorised orphan indication:

Treatment of metastatic adenocarcinoma of the pancreas, in combination with 5 fluorouracil (5 FU) and leucovorin (LV), in adult patients who have progressed following gemcitabine based therapy.

Orphan market exclusivity expiry date: 18 October 2026

GB Orphan designation number: PLGB 05815/0111/OD1

Onpattro

Active substance: patisiran

Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA

Orphan condition: Transthyretin-mediated amyloidosis

Authorised orphan indication:

Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.

Orphan market exclusivity expiry date: 29 August 2028

GB Orphan designation number: PLGB 50597/0002/OD1

Opfolda

Active substance: MIGLUSTAT

Orphan condition: Pompe’s disease adult onset

Authorised orphan indication: Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).

Orphan market exclusivity expiry date: 8 August 2033

GB Orphan Designation Number: PLGB 25823/0004/OD1

OXBRYTA

Active substance: voxelotor

Orphan condition: sickle cell disease

Authorised orphan indication: Oxbryta is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide.

Orphan market exclusivity date: 21 July 2032

GB Orphan Designation Number: PLGB 54981/0001/OD1

OXERVATE

Active substance: cenegermin

Orphan condition: neurotrophic keratitis

Authorised orphan indication: Treatment of moderate (persistent epithelial defect) or severe (corneal ulcer) neurotrophic keratitis in adults.

Orphan market exclusivity date: 10 July 2027

GB Orphan Designation Number: PLGB 32162/0002/OD1

Oxlumo

Active substance: lumasiran

Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues

Orphan condition: Primary hyperoxaluria

Authorised orphan indication:

Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups.

Orphan market exclusivity expiry date: 23 November 2030

GB Orphan designation number: PLGB 43942/0003/OD1

Palynziq

Active substance: pegvaliase

Orphan condition: hyperphenylalaninaemia

Authorised orphan indication:

Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.

Orphan market exclusivity date: 8 May 2029

GB Orphan Designation Number: PLGB 45814/0008 – 0010/OD1

Pemazyre

Active substance: pemigatinib

Orphan condition: Biliary tract cancer

Authorised orphan indication: The treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Orphan market exclusivity expiry date: 7 April 2031

GB Orphan designation number: PLGB 42338/0008 - 0010/OD1

Pombiliti 105 mg powder for concentrate for solution for infusion

Active substance: CIPAGLUCOSIDASE ALFA

Orphan condition: Glycogen storage disease type II (Pompe’s disease)

Authorised orphan indication: Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency

Orphan market exclusivity expiry date: 30 June 2033

GB Orphan designation number: PLGB 25823/0003/OD1

Polivy

Active substance: polatuzumab vedotin

Orphan condition: Diffuse large B-cell lymphoma

Authorised orphan indication:

Polivy in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (R-CHP) is indicated for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL).

Polivy in combination with bendamustine and rituximab is indicated for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.

Orphan market exclusivity expiry date: 20 January 2030

GB Orphan designation number: PLGB 00031/0912/OD1

Poteligeo

Active substance: mogamulizumab

Orphan condition: Cutaneous T-cell lymphoma

Authorised orphan indication:

POTELIGEO is indicated for the treatment of adult patients with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic therapy.

Orphan market exclusivity expiry date: 26 November 2028

GB Orphan designation number: PLGB 50262/0008/OD1

PREVYMIS

Active substance: letermovir (S)-{8-fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3,4-dihydro-4-quinazolinyl} acetic acid

Orphan condition: Cytomegalovirus disease reactivation in patients with impaired cell-mediated immunity

Authorised orphan indication:

PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT).

Consideration should be given to official guidance on the appropriate use of antiviral agents.

Orphan market exclusivity expiry date: 10 January 2028

GB Orphan designation number: PLGB 53095/0046 – 0048/OD1

PYRUKYND

Active substance: Mitapivat sulfate

Potent, allosteric activator of wild-type red blood cell (RBC)-specific form of pyruvate kinase (PKR) and a range of mutant PKR enzymes. Mitapivat targets the underlying enzymatic defect that causes haemolysis in pyruvate kinase deficiency by restoring the activity of mutant forms of PKR.

Orphan condition: pyruvate kinase deficiency (PK deficiency)

Authorised orphan indication:

Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients

Orphan market exclusivity expiry date: 29 November 2032

GB Orphan designation number: PLGB 52779/0001-0005OD1

Qarziba

Active substance: dinutuximab beta

Chimeric monoclonal antibody against GD2

Orphan condition: Neuroblastoma

Authorised orphan indication:

Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures.

In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin-2 (IL-2).

Orphan market exclusivity expiry date: 11 May 2027

GB Orphan designation number: PLGB 44185/0005/OD1

QINLOCK

Active substance: ripretinib

Orphan condition: Gastrointestinal stromal tumours

Authorised orphan indication: QINLOCK is indicated for the treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have received prior treatment with three or more kinase inhibitors, including imatinib

Orphan market exclusivity expiry date: 20 December 2031

GB Orphan Designation Number: PLGB 55295/0004/OD1

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Carbamoyl-phosphate synthase-1 deficiency

Authorised orphan indication:

Treatment of carbamoyl-phosphate synthase-1 deficiency:

RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD1

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Ornithine carbamoyltransferase deficiency

Authorised orphan indication:

Treatment of ornithine carbamoyltransferase deficiency:

RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD2

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Citrullinaemia type 1

Authorised orphan indication:

Treatment of citrullinaemia type 1:

RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD3

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Argininosuccinic aciduria

Authorised orphan indication:

Treatment of argininosuccinic aciduria:

RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.

RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD4

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Hyperargininaemia

Authorised orphan indication:

Treatment of hyperargininaemia:

RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD5

Ravicti

Active substance: Glycerol phenylbutyrate

Glyceryl tri-(4-phenylbutyrate)

Orphan condition: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)

Authorised orphan indication:

Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome): RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Orphan market exclusivity expiry date: 1 December 2027

GB Orphan designation number: PLGB 53487/0001/OD6

Raxone

Active substance: idebenone

Orphan condition: Leber’s hereditary optic neuropathy

Authorised orphan indication:

Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).

Orphan market exclusivity expiry date: 10 September 2025

GB Orphan designation number: PLGB 08829/0204/OD1

Reblozyl

Active substance: luspatercept

Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain

Orphan condition: Beta-thalassaemia intermedia and major

Authorised orphan indication: treatment of: - adult patients with transfusion-dependent anaemia associated with Beta-thalassaemia.

Orphan market exclusivity expiry date: 26 June 2030

GB Orphan designation number: PLGB 50412/0020 – 0021/OD1

Reblozyl

Active substance: luspatercept

Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain

Orphan condition: Myelodysplastic syndromes

Authorised orphan indication:

Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy

Orphan market exclusivity expiry date: 26 June 2030

GB Orphan designation number: PLGB 50412/0020 – 0021/OD2

REZUROCK

Active substance: belumosudil mesilate

Orphan condition: Graft versus host disease (GVHD)

Authorised orphan indication:

Rezurock is indicated for the treatment of patients aged 12 years and older with chronic graft-versus-host disease (chronic GVHD) who have received at least two prior lines of systemic therapy.

Orphan market exclusivity date:  6 July 2032

GB Orphan Designation Number: PLGB 53904/0001/OD1

Rezzayo

Active substance: REZAFUNGIN ACETATE  

Orphan condition: Treatment of invasive candidiasis

Authorised orphan indication:  Treatment of invasive candidiasis in adult

Orphan market exclusivity expiry date: 29/01/2034

GB Orphan Designation Number: PLGB 16950/0390/OD1

Rydapt

Active substance: midostaurin

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

Rydapt is indicated in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive

Orphan market exclusivity expiry date: 20 September 2027

GB Orphan designation number: PLGB 00101/1130/OD1

Rystiggo 140 mg/ml solution for injection  

Active substance: ROZANOLIXIZUMAB

Orphan condition: Myasthenia gravis

Authorised orphan indication: as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Orphan market exclusivity expiry date: 07/03/2034

GB Orphan designation number: PLGB 00039/0808/OD1

Rydapt

Active substance: midostaurin

Orphan condition: Mastocytosis

Authorised orphan indication:

Rydapt is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL).

Orphan market exclusivity expiry date: 20 September 2027

GB Orphan designation number: PLGB 00101/1130/OD2

SCENESSE

Active substance: afamelanotide

[Nle4, D-Phe7]-alpha-melanocyte stimulating hormone

Orphan condition: Erythropoietic protoporphyria

Authorised orphan indication:

Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).

Orphan market exclusivity expiry date: 29 December 2024

GB Orphan designation number: PLGB 30619/0002/OD1

Scemblix

Active substance: Asciminib

Orphan condition: Chronic myeloid leukaemia

Authorised orphan indication:

Scemblix is indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation

Orphan market exclusivity expiry date: 15 June 2032

GB Orphan designation number: PLGB 00101/1207/OD1

Sogroya

Active substance: somapacitan

Orphan condition: Growth hormone deficiency

Authorised orphan indication:

Sogroya is indicated for the replacement of endogenous growth hormone (GH) in children aged 3 years and above, and adolescents with growth failure due to growth hormone deficiency (paediatric GHD), and in adults with growth hormone deficiency (adult GHD).

Orphan market exclusivity expiry date: 21 October 2031

GB Orphan Designation Number: PLGB 04668/0434/OD1

Soliris

Active substance: eculizumab

Orphan condition: Myasthenia gravis

Authorised orphan indication:

Soliris is indicated for the treatment of refractory generalized myasthenia gravis (gMG) in patients aged 6 years and above who are antiacetylcholine receptor (AChR) antibody-positive

Orphan market exclusivity expiry date: 17 August 2027

GB Orphan designation number: PLGB 31775/0003/OD2

Soliris

Active substance: eculizumab

Orphan condition: Neuromyelitis optica spectrum disorders

Authorised orphan indication:

Soliris is indicated in adults for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease.

Orphan market exclusivity expiry date: 28 August 2029

GB Orphan designation number: PLGB 31775/0003/OD3

SomaKit TOC

Active substance: edotreotide

Gallium (68Ga)-edotreotide

Orphan condition: Gastro-entero-pancreatic neuroendocrine tumours

Authorised orphan indication:

This medicinal product is for diagnostic use only.

After radiolabelling with gallium (68Ga) chloride solution, the solution of gallium (68Ga) edotreotide obtained is indicated for Positron Emission Tomography (PET) imaging of somatostatin receptor overexpression in adult patients with confirmed or suspected well-differentiated gastro-enteropancreatic neuroendocrine tumours (GEP-NET) for localizing primary tumours and their metastases.

Orphan market exclusivity expiry date: 12 December 2026

GB Orphan designation number: PLGB 35145/0004/OD1

Spexotras

Active substance: Trametinib

Orphan condition: Glioma

Authorised orphan indication:

Low-grade glioma

Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy.

High-grade glioma

Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

Orphan market exclusivity expiry date: 13 February 2036

GB Orphan designation number: PLGB 00101/1227/OD1

Spinraza

Active substance: nusinersen

Antisense oligonucleotide targeted to the SMN2 gene

Orphan condition: 5q spinal muscular atrophy

Authorised orphan indication:

Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.

Orphan market exclusivity expiry date: 1 June 2029

GB Orphan designation number: PLGB 22407/0018/OD1

Strensiq

Active substance: asfotase alfa

Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein

Orphan condition: Hypophosphatasia

Authorised orphan indication:

Strensiq is indicated for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia to treat the bone manifestations of the disease.

Orphan market exclusivity expiry date: 1 September 2025

GB Orphan designation number: PLGB 31775/0004 – 0005/OD1

Strimvelis

Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence

Orphan condition: Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency

Authorised orphan indication:

Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

Orphan market exclusivity expiry date: 30 May 2028

GB Orphan designation number: PLGB 49055/0001/OD1

Symkevi

Active substance: tezacaftor/ivacaftor

Orphan condition: Cystic fibrosis

1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor

Authorised orphan indication:

Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T.

Orphan market exclusivity expiry date: 6 November 2028

GB Orphan designation number: PLGB 22352/0003/OD1

TAKHZYRO

Active substance: lanadelumab

Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein

Orphan condition: Hereditary angioedema

Authorised orphan indication:

TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older.

Orphan market exclusivity expiry date: 26 November 2030

GB Orphan designation number: PLGB 54937/0017/OD1

TAVNEOS

Active substance: tavacopan

Orphan condition: Granulomatosis with polyangiitis

Authorised orphan indication: Tavneos, In combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

Orphan market exclusivity expiry date: 5 May 2032

GB Orphan Designation Number: PLGB 50784/0008/OD1

TAVNEOS

Active substance: tavacopan

Orphan condition: Microscopic polyangiitis

Authorised orphan indication: Tavneos, In combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

Orphan market exclusivity expiry date: 5 May 2032

GB Orphan Designation Number: PLGB 50784/0008/OD2

Tecartus

Active substance: autologous anti-CD19-transduced CD3+ cells

Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured

Orphan condition: Acute lymphoblastic leukaemia

Authorised orphan indication:

Tecartus is indicated for the treatment of adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

Orphan market exclusivity expiry date: 7 November 2032

GB Orphan designation number: PLGB 11972/0045/OD2

Tecartus

Active substance: autologous anti-CD19-transduced CD3+ cells

Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured

Orphan condition: Mantle cell lymphoma

Authorised orphan indication:

Tecartus is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor.

Orphan market exclusivity expiry date: 14 December 2030

GB Orphan designation number: PLGB 11972/0045/OD1

Tegsedi

Active substance: inotersen

Phosphorothioate oligonucleotide targeted to transthyretin

Orphan condition: ATTR amyloidosis

Authorised orphan indication:

Tegsedi is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

Orphan market exclusivity expiry date: 10 July 2028

GB Orphan designation number: PLGB 51704/0002/OD1

TIBSOVO

Active substance: ivosidenib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication: In combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy (see section 5.1)

Orphan market exclusivity expiry date: 5 July 2033

GB Orphan Designation Number: PLGB 05815/0120/OD1

TIBSOVO

Active substance:  ivosidenib

Orphan condition: Biliary tract cancer

Authorised orphan indication: as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (see section 5.1).

Orphan market exclusivity expiry date: 5 July 2033

GB Orphan Designation Number: PLGB 05815/0120/OD2

Trecondi

Active substance: treosulfan

Orphan condition: Haematopoietic progenitor cell transplantation

Authorised orphan indication:

Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non malignant diseases.

Orphan market exclusivity expiry date: 24 June 2031

GB Orphan designation number: PLGB 11587/0118 - 0119/OD1

Trepulmix

Active substance: treprostinil sodium

Orphan condition: Chronic thromboembolic pulmonary hypertension

Authorised orphan indication:

Trepulmix is indicated for the treatment of adult patients with WHO Functional Class (FC) III or IV and:

• inoperable chronic thromboembolic pulmonary hypertension (CTEPH), or
• persistent or recurrent CTEPH after surgical treatment to improve exercise capacity.

Orphan market exclusivity expiry date: 7 April 2030

GB Orphan designation number: PLGB 51174/0001 – 0004/OD1

UPSTAZA

Active substance: eladocagene exuparvovec

Orphan condition: Aromatic L-amino acid decarboxylase deficiency

Authorised orphan indication:

For the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Orphan market exclusivity expiry date: 17 November 2034

GB Orphan designation number: PLGB 44221/0006/OD1V

Vanflyta

Active substance: Quizartinib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication: In combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD positive

Orphan market exclusivity expiry date: 11/03/2034

GB Orphan Designation Number: PLGB 08265/0047-0048/OD1

Verkazia

Active substance: ciclosporin

Orphan condition: Vernal keratoconjunctivitis

Authorised orphan indication:

Treatment of severe vernal keratoconjunctivitis (VKC) in children from 4 years of age and adolescents

Orphan market exclusivity expiry date: 10 July 2030

GB Orphan designation number: PLGB 16508/0028/OD1

Voraxaze 1000 units powder for solution for injection

Active substance: GLUCARPIDASE

Orphan condition: Methotrexate-induced nephropathy

Authorised orphan indication: Voraxaze is indicated to reduce toxic plasma methotrexate concentration in adults and children (aged 28 days and older) with delayed methotrexate elimination.

Orphan market exclusivity expiry date: 15 June 2035

GB Orphan designation number: PLGB 18442/0002/OD1

Vyloy

Active substance: zolbetuximab

Orphan condition: malignant neoplasm of stomach

Authorised orphan indication: Vyloy, in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 -negative gastric or gastro-oesophageal junction adenocarcinoma whose tumours are Claudin 18.2 positive

Orphan market exclusivity expiry date: 14/08/2034

GB Orphan Designation Number: PLGB 00166/0439/OD1

Vyndaqel

Active substance: tafamidis

N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate

Orphan condition: Senile systemic amyloidosis

Authorised orphan indication:

treatment of wild-type transthyretin amyloidosis in adult patients with cardiomyopathy

Orphan market exclusivity expiry date: 19 February 2030

GB Orphan designation number: PLGB 00057/1660/OD2

Note: Wild-type transthyretin amyloid is also known as senile systemic amyloidosis and Hereditary transthyretin amyloid is also known as familial amyloidosis. The protection for hereditary amyloid ended on 18 Nov 2021.

Vyvgart 20 mg/ml Concentrate for solution for infusion

Active substance: EFGARTIGIMOD ALFA

Orphan condition: Myasthenia Gravis (gMG)

Authorised orphan indication:

Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

Orphan market exclusivity expiry date: 14 March 2033

GB Orphan designation number: PLGB 47104/0004/OD1Vyvgart 20 mg/ml Concentrate for solution for infusion

Active substance: EFGARTIGIMOD ALFA

Orphan condition: Myasthenia Gravis (gMG)

Authorised orphan indication:

Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.

Orphan market exclusivity expiry date: 14 March 2033

GB Orphan designation number: PLGB 47104/0004/OD1

Vyxeos liposomal

Active substance: daunorubicin / cytarabine

Liposomal combination of cytarabine and daunorubicin

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

Vyxeos is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 31626/0004/OD1

Wainzua

Wainzua 45 mg solution for injection in pre-filled pen

Active substance: EPLONTERSEN

Orphan condition: Transthyretin-mediated amyloidosis

Authorised orphan indication: Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy.

Orphan market exclusivity expiry date: 14 October 2034

GB Orphan designation number: PLGB 17901/0377/OD1

Welireg

Active substance: BELZUTIFAN

Orphan condition: von Hippel-Lindau (VHL) disease

Authorised orphan indication:

Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable.

Orphan market exclusivity expiry date: 30 May 2032

GB Orphan designation number: PLGB 53095/0087/OD1

XENPOZYME

Active substance: Olipudase alfa

Orphan condition: Acid sphingomyelinase deficiency

Authorised orphan indication:

Enzyme replacement therapy for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency in paediatric and adult patients with type A/B or type B.

Orphan market exclusivity expiry date: 31 July 2032

GB Orphan designation number: PLGB 04425/0901/OD1

Waylivra

Active substance: volanesorsen

Phosphorothioate oligonucleotide targeted to apolipoprotein C-III

Orphan condition: Familial chylomicronemia syndrome

Authorised orphan indication:

Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate

Orphan market exclusivity expiry date: 8 May 2029

GB Orphan designation number: PLGB 51704/0003/OD1

Xermelo

Active substance: telotristat

(S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate

Orphan condition: Carcinoid syndrome

Authorised orphan indication:

Xermelo is indicated for the treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy.

Orphan market exclusivity expiry date: 20 September 2027

GB Orphan designation number: PLGB 28247/0008/OD1

Xospata

Active substance: gilteritinib

Orphan condition: Acute myeloid leukaemia

Authorised orphan indication:

Xospata is indicated as monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation.

Orphan market exclusivity expiry date: 28 October 2029

GB Orphan designation number: PLGB 00166/0425/OD1

Yescarta

Active substance: axicabtagene ciloleucel

Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor

Orphan condition: Diffuse large B cell lymphoma

Authorised orphan indication:

treatment of adult patients with diffuse large B cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy.

treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 11972/0044/OD1

Yescarta

Active substance: axicabtagene ciloleucel

Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor

Orphan condition: Primary mediastinal large B-cell lymphoma

Authorised orphan indication:

YESCARTA is indicated for the treatment of adult patients with relapsed or refractory primary mediastinal large B-cell lymphoma (PMBCL) after two or more lines of systemic therapy.

Orphan market exclusivity expiry date: 27 August 2028

GB Orphan designation number: PLGB 11972/0044/OD2

Yescarta

Active substance: axicabtagene ciloleucel

Orphan condition: Follicular lymphoma

Authorised orphan indication: treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy’

Orphan market exclusivity expiry date: 9 August 2032

GB Orphan Designation Number: PLGB 11972/0044/OD3

Yorvipath 168 micrograms/0.56 mL solution for injection in pre filled pen

Active substance: PALOPEGTERIPARATIDE 

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Yorvipath 294 micrograms/0.98 mL solution for injection in pre filled pen

Active substance: PALOPEGTERIPARATIDE 

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Yorvipath 420 micrograms/1.4 mL solution for injection in pre filled pen

Active substance: PALOPEGTERIPARATIDE 

Orphan condition: Hypoparathyroidism

Authorised orphan indication: Treatment of hypoparathyroidism.

Orphan market exclusivity expiry date: 23/04/2034

GB Orphan designation number: PLGB 51127/0001-0003/OD1

Zejula

Active substance: niraparib

(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt

Orphan condition: Ovarian cancer

Authorised orphan indication:

Zejula is indicated:

• as monotherapy for the maintenance treatment of adult patients with advanced epithelial (FIGO Stages III and IV) high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy.
• as monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum based chemotherapy

Orphan market exclusivity expiry date: 20 November 2027

GB Orphan designation number: PLGB 19494/0294/OD1

Zokinvy

Active substance: lonafarnib

Orphan condition: Hutchinson-Gilford progeria syndrome

Authorised orphan indication:

Treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.

Orphan market exclusivity expiry date: 24 August 2034

GB Orphan designation number: PLGB 56709/0001-0002/ OD1

Zolgensma

Active substance: onasemnogene abeparvovec

Adeno-associated viral vector serotype 9 containing the human SMN gene

Orphan condition:

Spinal muscular atrophy

Authorised orphan indication:

Zolgensma is indicated for the treatment of:

• patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or
• patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

Orphan market exclusivity expiry date: 18 May 2030

GB Orphan designation number: PLGB 53139/0002/OD1

Ztalmy 50 mg/ml Oral suspension  

Active substance: GANAXOLONE  

Orphan condition: Cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder

Authorised orphan indication: indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

Orphan market exclusivity expiry date: 07/03/2034

GB Orphan designation number: PLGB 55682/0001/OD1

Zynteglo

Active substance: betibeglogene autotemcel

Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene

Orphan condition: Beta-thalassaemia intermedia and major

Authorised orphan indication:

Zynteglo is indicated for the treatment of patients 12 years and older with transfusion dependent β thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA) matched related HSC donor is not available

Orphan market exclusivity expiry date: 4 June 2029

GB Orphan designation number: PLGB 51370/0001/OD1