Expired orphan registrations
Updated 8 November 2024
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A list of orphan authorisations previously registered by the UK Licensing Authority where the orphan market exclusivity period has ended.
ADCETRIS
Active substance: brentuximab vedotin
Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
Orphan condition: Systemic anaplastic large cell lymphoma.
Authorised orphan indication:
ADCETRIS in combination with cyclophosphamide, doxorubicin and prednisone (CHP) is indicated for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL).
ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory sALCL.
Orphan market exclusivity expiry date: 30 October 2024
GB Orphan designation number: PLGB 16189/0093/OD1
ADCETRIS
Active substance: brentuximab vedotin
Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E
Orphan condition: Hodgkin lymphoma
Authorised orphan indication:
ADCETRIS is indicated for adult patients with previously untreated CD30+ Stage IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD).
ADCETRIS is indicated for the treatment of adult patients with CD30+ HL at increased risk of relapse or progression following autologous stem cell transplant (ASCT).
ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL):
- following ASCT, or
- following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option.
Orphan market exclusivity expiry date: 30 October 2024
GB Orphan designation number: PLGB 16189/0093/OD2
Adempas
Active substance: riociguat
Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate
Orphan condition: Chronic thromboembolic pulmonary hypertension (CTEPH)
Authorised orphan indication:
Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with
- inoperable CTEPH
- persistent or recurrent CTEPH after surgical treatment, to improve exercise capacity.
Pulmonary arterial hypertension (PAH)
Adempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity.
Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease.
Orphan market exclusivity expiry date: 31 Mar 2024
GB Orphan designation number: PLGB 00010/0670 – 0674/OD1
Bronchitol
Active substance: Mannitolum
Orphan condition: Cystic fibrosis
Authorised orphan indication:
Bronchitol is indicated for the treatment of cystic fibrosis (CF) in adults aged 18 years and above as an add-on therapy to best standard of care.
Orphan market exclusivity expiry date: 18 Apr 2022
GB Orphan designation number: PLGB 27944/0002/OD1
Carbaglu
Active substance: Carglumic acid
Orphan condition: Isovaleric acidaemia
Authorised orphan indication:
Carbaglu is indicated in treatment of hyperammonaemia due to isovaleric acidaemia.
Orphan market exclusivity expiry date: 1 Jun 2021
GB Orphan designation number: PLGB 15266/0019/OD1
Carbaglu
Active substance: Carglumic acid
Orphan condition: Methylmalonic acidaemia
Authorised orphan indication:
Carbaglu is indicated in treatment of hyperammonaemia due to methymalonic acidaemia.
Orphan market exclusivity expiry date: 1 Jun 2021
GB Orphan designation number: PLGB 15266/0019/OD2
Carbaglu
Active substance: Carglumic acid
Orphan condition: Propionic acidaemia
Authorised orphan indication:
Carbaglu is indicated in treatment of hyperammonaemia due to propionic acidaemia.
Orphan market exclusivity expiry date: 1 Jun 2021
GB Orphan designation number: PLGB 15266/0019/OD3
Cometriq
Active substance: cabozantinib
Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt
Orphan condition: Medullary thyroid carcinoma
Authorised orphan indication:
COMETRIQ is indicated for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma.
For patients in whom rearranged during transfection (RET) mutation status is not known or is negative, a possible lower benefit should be taken into account before individual treatment decision.
Orphan market exclusivity expiry date: 26 March 2024
GB Orphan designation number: PLGB 28247/0004 – 0005/OD1
Dacogen
Active substance: decitabine
Orphan condition: Acute myeloid leukaemia
Authorised orphan indication:
Dacogen is indicated for the treatment of adult patients with newly diagnosed de novo or secondary acute myeloid leukaemia (AML), according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy.
Orphan market exclusivity expiry date: 24 September 2024
GB Orphan designation number: PLGB 00242/0662/OD1
Defitelio
Active substance: defibrotide
Orphan condition: Hepatic veno-occlusive disease
Authorised orphan indication:
Treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.
It is indicated in adults and in adolescents, children and infants over 1 month of age.
Orphan market exclusivity expiry date: 22 Oct 2023
GB Orphan designation number: PLGB 31626/0005/OD1
Esbriet
Active substance: pirfenidone
Orphan condition: Idiopathic pulmonary fibrosis
Authorised orphan indication:
Esbriet is indicated in adults for the treatment of mild to moderate Idiopathic Pulmonary Fibrosis (IPF).
Orphan market exclusivity expiry date: 2 Mar 2021
GB Orphan designation number: PLGB 00031/0851 – 0854/OD1
Evrysdi
Active substance: risdiplam
Orphan condition: Spinal Muscular Atrophy
Authorised orphan indication:
Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
Orphan market exclusivity expiry date: 19 May 2031
GB Orphan designation number: PLGB 00031/0920/OD1
Gazyvaro
Active substance: obinutuzumab
Orphan condition: Chronic lymphocytic leukaemia (CLL)
Authorised orphan indication:
Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated CLL and with comorbidities making them unsuitable for full-dose fludarabine based therapy (see section 5.1).
Orphan market exclusivity expiry date: 24 July 2024
GB Orphan designation number: PLGB 00031/0856/OD1
Granupas
Active substance: Para-aminosalicylic acid
Orphan condition: Tuberculosis
Authorised orphan indication:
Indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients from 28 days of age and older when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.
Consideration should be given to official guidance on the appropriate use of antibacterial agents.
Orphan market exclusivity expiry date: 9 April 2024
GB Orphan designation number: PLGB 35068/0003/OD1
Hepcludex
Active substance: bulevirtide
Orphan condition: Hepatitis delta virus infection
Authorised orphan indication:
The treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDV-RNA positive adult patients with compensated liver disease.
Orphan market exclusivity expiry date: 05 August 2030
GB Orphan designation number: PLGB 50662/0002/OD1
Iclusig
Active substance: ponatinib
Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl]
Orphan condition: Acute lymphoblastic leukaemia
Authorised orphan indication:
Iclusig is indicated in adult patients with
Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
Orphan market exclusivity expiry date: 3 Jul 2023
GB Orphan designation number: PLGB 49635/0001 – 0003/OD1
Iclusig
Active substance: ponatinib
Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl]
Orphan condition: Chronic myeloid leukaemia
Authorised orphan indication:
Iclusig is indicated in adult patients with
- chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation
Orphan market exclusivity expiry date: 3 Jul 2023
GB Orphan designation number: PLGB 49635/0001 – 0003/OD2
Imnovid
Active substance: pomalidomide
Orphan condition: Multiple myeloma
Authorised orphan indication:
Imnovid in combination with dexamethasone is indicated in the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy
Orphan market exclusivity expiry date: 8 Aug 2023
GB Orphan designation number: PLGB 50412/0002 – 0005/OD1
Kalydeco
Active substance: ivacaftor
N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide
Orphan condition: Cystic fibrosis
Authorised orphan indication:
Treatment of cystic fibrosis (CF) in patients age six years and older who have a G551D mutation in the CFTR gene
Orphan market exclusivity expiry date: 25 July 2022
GB Orphan designation number: PLGB 22352/0008 – 0011/OD1
Kalydeco
Active substance: ivacaftor
N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide
Orphan condition: Cystic fibrosis
Authorised orphan indication:
In a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1)
Orphan market exclusivity expiry date: 25 July 2022
GB Orphan designation number: PLGB 22352/0008/OD1
GB Orphan Designation number: PLGB 22352/0013/OD1
Mozobil
Active substance: plerixafor
l, 1’-[1,4-phenylenebis (methylene)]-bis-1,4,8,11- tetraazacyclotetradecane
Orphan condition: Mobilization of progenitor cells prior to stem cell transplantation
Authorised orphan indication:
Adult patients
Mozobil is indicated in combination with granulocyte-colony stimulating factor (G-CSF) to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in adult patients with lymphoma or multiple myeloma whose cells mobilise poorly.
Paediatric patients (1 to less than 18 years)
Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either:
-
pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or
-
who previously failed to collect sufficient haematopoietic stem cells.
Orphan market exclusivity expiry date: 4 Aug 2021
GB Orphan designation number: PLGB 04425/0769/OD1
Nexavar
Active substance: sorafenib tosylate
Orphan condition: Follicular thyroid cancer
Authorised orphan indication:
Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (follicular/Hürthle cell) thyroid carcinoma, refractory to radioactive iodine.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 00010/0701/OD1
Nexavar
Active substance: sorafenib tosylate
Orphan condition: Papillary thyroid cancer
Authorised orphan indication:
Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (papillary) thyroid carcinoma, refractory to radioactive iodine.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 00010/0701/OD2
NexoBrid
Active substance: Concentrate of proteolytic enzymes enriched in bromelain
Purified bromelain
Orphan condition:
Partial deep dermal and full thickness burns
Authorised orphan indication:
NexoBrid is indicated for removal of eschar in adults with deep partial- and full-thickness thermal burns.
Orphan market exclusivity expiry date: 20 Dec 2022
GB Orphan designation number: PLGB 43948/0002 – 0003/OD1
Opsumit
Active substance: macitentan
Orphan condition: Pulmonary arterial hypertension
Authorised orphan indication:
Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III.
Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.
Orphan market exclusivity expiry date: 27 December 2023
GB Orphan designation number: PLGB 00242/0663/OD1
Plenadren
Active substance: hydrocortisone
Orphan condition: Adrenal insufficiency
Authorised orphan indication:
Treatment of adrenal insufficiency in adults.
Orphan market exclusivity expiry date: 14 Nov 2021
GB Orphan designation number: PLGB 39534/0009-0010/OD1
Revestive
Active substance: teduglutide
[gly2] Recombinant human glucagon-like peptide
Orphan condition: Short Bowel Syndrome
Authorised orphan indication:
Revestive is indicated for the treatment of patients aged 1 year and above with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery.
Orphan market exclusivity expiry date: 4 September 2024
GB Orphan designation number: PLGB 16189/0077 – 0078/OD1
Signifor
Active substance: pasireotide
Orphan condition: Cushing’s disease
Authorised orphan indication:
Treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed.
Orphan market exclusivity expiry date: 27 Apr 2022
GB Orphan designation number: PLGB 15266/0032 – 0039/OD2
Sirturo 20 mg Tablets
Active substance: BEDAQUILINE FUMARATE
Orphan condition: tuberculosis
Authorised orphan indication: as per section 4.1 of the SPC
Orphan market exclusivity expiry date: 7 March 2024
GB Orphan designation number: PLGB 00242/0744 /OD1
Soliris
Active substance: eculizumab
Orphan condition: Atypical haemolytic uremic syndrome
Authorised orphan indication:
Soliris is indicated in adults and children for the treatment of atypical haemolytic uremic syndrome (aHUS).
Orphan market exclusivity expiry date: 29 November 2023
GB Orphan designation number: PLGB 31775/0003/OD1
Sylvant
Active substance: siltuximab
Chimeric-anti-interleukin-6 monoclonal antibody
Orphan condition: Castleman’s disease
Authorised orphan indication:
Sylvant is indicated for the treatment of adult patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus 8 (HHV 8) negative.
Orphan market exclusivity expiry date: 27 May 2024
GB Orphan designation number: PLGB 44185/0006 – 0007/OD1
TOBI Podhaler
Active substance: tobramycin
Orphan condition: Pseudomonas aeruginosa lung infection in cystic fibrosis
Authorised orphan indication:
TOBI Podhaler is indicated for the suppressive therapy of chronic pulmonary infection due to Pseudomonas aeruginosa in adults and children aged 6 years and older with cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents.
Orphan market exclusivity expiry date: 25 Jul 2023
GB Orphan designation number: PLGB 46302/0222/OD1
Translarna
Active substance: ataluren
(3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid
Orphan condition: Duchenne muscular dystrophy
Authorised orphan indication:
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing
Orphan market exclusivity expiry date: 5 August 2024
GB Orphan designation number: PLGB 44221/0002 – 0004/OD1
Vimizim
Active substance: elosulfase alfa
Recombinant human N-acetylgalactosamine-6-sulfatase
Orphan condition: Mucopolysaccharidosis, type IVA (Morquio A Syndrome)
Authorised orphan indication:
Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.
Orphan market exclusivity expiry date: 30 April 2024
GB Orphan designation number: PLGB 45814/0007/OD1
VPRIV
Active substance: velaglucerase alfa
Orphan condition: Gaucher disease
Authorised orphan indication:
VPRIV is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease.
Orphan market exclusivity expiry date: 30 Aug 2022
GB Orphan designation number: PLGB 16189/0088/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: Familial amyloid polyneuropathy
Authorised orphan indication:
Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment.
Orphan market exclusivity expiry date: 18 Nov 2021
GB Orphan designation number: PLGB 00057/1660/OD1
Xaluprine
Active substance: mercaptopurine
Mercaptopurine (oral suspension)
Orphan condition:
Acute lymphoblastic leukaemia
Authorised orphan indication:
Treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children.
Orphan market exclusivity expiry date: 13 Mar 2022
GB Orphan designation number: PLGB 13581/0002/OD1