Decision

Orphan registered medicinal products

A list of authorised orphan medicinal products registered by the UK licensing authority.

From:
Medicines and Healthcare products Regulatory Agency
Published
9 February 2021
Last updated
23 December 2024 — See all updates

Documents

Orphan register

HTML

Expired orphan registrations

HTML

Details

The orphan register comprises:

  • EU marketing authorisations converted into Great Britain marketing authorisations in accordance with paragraph 6(7) of Schedule 33A the Human Medicines Regulations 2012 (as amended) where there is an existing European Union (EU) orphan designation, and the Great Britain marketing authorisation continues in effect with the remaining period of orphan market exclusivity
  • medicinal products that have received a marketing authorisation with orphan status on or after 1 January 2021 from MHRA, the UK licensing authority

Under regulation 58D of the Human Medicines Regulations 2012 (as amended), a period of 10 years orphan market exclusivity is awarded from the date of marketing authorisation by MHRA. An additional 2 years of exclusivity may be added where paediatric data requirements have been met.

This is in accordance with regulation 58C(2) of the Human Medicines Regulations 2012 (as amended).

The scope of orphan market exclusivity is defined by the authorised indication.

Products with an orphan designation in the EU can be considered for a Great Britain orphan marketing authorisation.

A UK-wide orphan marketing authorisation can only be considered in the absence of an active EU orphan designation.

There is no pre-authorisation orphan designation in Great Britain.

An orphan designation effective in Great Britain or UK is reflected in PLGB or PL, respectively, in the orphan designation number.

Updates to this page

Published 9 February 2021
Last updated 23 December 2024 + show all updates

  1. Added Givinostat - PLGB 13297/0017 to Orphan Register

  2. Removed Wakix from orphan register as product has been withdrawn

  3. Updated orphan register for BLINCYTO

  4. Added FINLEE, Active substance: DABRAFENIB MESYLATE to Orphan Register

  5. Added FABHALTA to Orphan Register

  6. Added - 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.

  7. ADCETRIS (PLGB 16189/0093/OD1) & (PLGB 16189/0093/OD2) removed from Orphan Register and added to Expired Orphan Registrations.

  8. Added 'IQIRVO' and 'Wainzua' to the Orphan Register.

  9. Added Joenja to orphan register

  10. Dacogen (PLGB 00242/0662/OD1) added to expired list.

  11. Changed the Orphan market exclusivity expiry date of Fintepla on the Orphan Register to 5 July 2033.

  12. Removed Revestive from orphan registered list and placed in to Expired orphan registrations

  13. Amended to change expiry date on orphan registration of Agamree

  14. Changed Orphan Register to add Vyloy (Active substance: zolbetuximab)

  15. Added to remove Translarna from Orphan products to expired orphan products

  16. Gazyvaro moved to 'Expired orphan registrations'

  17. Updated exclusivity expiry date for Trecondi to 24 June 2031 & moved Vimizim to the 'expired' list.

  18. Updated information in the authorised orphan indication for Carvykti.

  19. Moved Cometriq from the orphan register to the expired orphan register

  20. Yorvipath 168, 294 and 420 micrograms added to the orphan register

  21. Granupas added to the expired orphan register.

  22. Updated information in the authorised orphan indication for TAKHZYRO.

  23. Rystiggo and Ztalmy added to the orphan register

  24. Updated to add AGAMREE to orphan register, updated indication of Soliris, removed Sirturo 20 mg Tablets from orphan register and added to expired orphan registrations

  25. Updated Orphan Register with Vanflyta

  26. The day or market exclusivity for Spexotras has been updated to 13 February 2036 in the Orphan Register.

  27. Spexotras has been added to the Orphan register

  28. Moved Imnovid, Opsumit, & Soliris from Orphan Register, to the Expired Orphan register.

  29. Moved 'Hepcludex' from 'Orphan Register' to 'Expired orphan registrations' list.

  30. Added 'Loargys 5 mg/ml solution for injection/infusion' to the orphan register

  31. Added 'Enspryng' to the orphan register

  32. Orphan register updated with new entries: Casgevy for β thalassaemia intermedia and major and Casgevy for sickle cell disease

  33. Orphan register updated with new entry: Hyftor.

  34. Updated Orphan register removing Defitelio and adding it to the expired register list.

  35. 'Evrysdi' moved to Expired Orphan Register.

  36. 'Columvi 2.5 mg and 10mg concentrate for solution for infusion' added to Orphan register.

  37. Updated Orphan Register to update Orphan market exclusivity date on the Imcivree injection 10mg/ml to 17 November 2032

  38. Added 'ELZONRIS' to Orphan Register.

  39. TOBI Podhale entry removed from the Orphan register.

  40. Added Tecartus for mantle cell lymphoma to the orphan register

  41. Orphan register updated to include TIBSOVO

  42. Added 'Pombiliti 105 mg powder for concentrate for solution for infusion' to the Orphan Register.

  43. Iclusig removed from the Orphan register.

  44. Updated Orphan Register - added Voraxaze 1000 units powder for solution for injection

  45. Added 'Ebvallo 2.8 x 10^7 - 7.3 x 10^7 cells/mL dispersion for injection ' to Orphan register.

  46. Added 'Nexviadyme 100 mg powder for concentrate for solution for infusion' to the Orphan Register.

  47. Moved 'Adempas' to Expired orphan registrations

  48. Orphan register updated

  49. Added 'Vyvgart 20 mg/ml Concentrate for solution for infusion' to the orphan register

  50. Added 'Kinpeygo - Active substance: Budesonide' to the orphan register.

  51. Added Carvykti to the orphan register

  52. Update to 'Orphan Register' & 'Expired orphan registrations'

  53. Addition of Yescarta for the 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

  54. New orphan product UPSTAZA added to the orphan register.

  55. Tecartus section in the Orphan register updated.

  56. Lonapegsomatropin Ascendis Pharma has been added to the Orphan Register.

  57. Added 2 entries for TAVNEOS to the Orphan Register

  58. Added Lunsumio to Orphan list

  59. Artesunate Amivas has been added to the Orphan Register

  60. Orphan and Expired-orphan registers updated.

  61. Added Zokinvy to Orphan register.

  62. Added 'Rezurock' as a new entry to the Orphan register.

  63. Added FILSUVEZ to Orphan Register

  64. Added Kymriah & Yescarta to Orphan Register

  65. XENPOZYME added to Orphan Register.

  66. Updated Revestive orphan market exclusivity expiry date, to 4 Sep 2024.

  67. Added OXBRYTA to Orphan Register

  68. Updated 'Authorised orphan indication' for Ngenla.

  69. Orphan indication updated for Polivy.

  70. Added ABECMA to the Orphan register.

  71. Scemblix added to Orphan register

  72. Added Kimmtrak to the Orphan Register

  73. Orphan market exclusivity expiry extended to 30 Oct 2024, for PLGB 16189/0093/OD2 & PLGB 16189/0093/OD1

  74. Ngenla added to Orphan Register

  75. Xaluprine moved from Live Orphan Register to Expired Orphan Register.

  76. A new product called Sogroya was added to the Orphan Register.

  77. Updated entries on the 'Orphan Register'

  78. Plenadren moved to expired list

  79. Updates to the entries for Kaftrio and Kalydeco on the Orphan Register

  80. QINLOCK added to the Orphan Register

  81. The orphan market exclusivity period for Vyndaqel has ended. Register has been updated.

  82. Imbruvica removed from register

  83. 'Minjuvi' has been added to the Orphan Register

  84. Addition of Darzalex for the treatment of Systemic light chain (AL) amyloidosis, Epidyolex for the treatment of Tuberous sclerosis complex and Bylvay for the treatment of Progressive familial intrahepatic cholestasis.

  85. Added Koselugo to the Orphan register

  86. Mozobil moved to expired orphan registration page

  87. Updated Orphan register with Palynziq and OXERVATE

  88. Newly added entry to Orphan Register - Hepcludex

  89. Moved Orphan registered products 'Carbaglu' from 'Orphan register' page to 'Expired orphan registrations' page.

  90. Added Adakveo to the Orphan Register list.

  91. An orphan marketing authorisation for Evrysdi 0.75 mg/mL powder for oral solution has been granted

  92. Edited the authorised orphan indication for Kaftrio, and added a second entry for Kalydeco. The second entry is reflected in a separate entry as it only concerns one strength of the product (this is reflected in the PLGB number).

  93. Added Inrebic to the Orphan Register list.

  94. Added Pemazyre to the Orphan Register

  95. Published a new HTML document, “Expired orphan registrations”. This is a list of orphan authorisations previously registered by the UK Licensing Authority where the orphan market exclusivity period has ended. The listing for Esbriet has expired so has been removed from the register and is now in the 'Expired' list.

  96. Added information on orphan conditions to the register entries

  97. First published.

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