Decision

Orphan registered medicinal products

A list of authorised orphan medicinal products registered by the UK Licensing Authority.

Documents

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 the UK Licensing Authority, the MHRA. 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 the MHRA. An additional two 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.

Published 9 February 2021
Last updated 1 March 2024 + show all updates
  1. The day or market exclusivity for Spexotras has been updated to 13 February 2036 in the Orphan Register.

  2. Spexotras has been added to the Orphan register

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

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

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

  6. Added 'Enspryng' to the orphan register

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

  8. Orphan register updated with new entry: Hyftor.

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

  10. 'Evrysdi' moved to Expired Orphan Register.

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

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

  13. Added 'ELZONRIS' to Orphan Register.

  14. TOBI Podhale entry removed from the Orphan register.

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

  16. Orphan register updated to include TIBSOVO

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

  18. Iclusig removed from the Orphan register.

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

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

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

  22. Moved 'Adempas' to Expired orphan registrations

  23. Orphan register updated

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

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

  26. Added Carvykti to the orphan register

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

  28. 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

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

  30. Tecartus section in the Orphan register updated.

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

  32. Added 2 entries for TAVNEOS to the Orphan Register

  33. Added Lunsumio to Orphan list

  34. Artesunate Amivas has been added to the Orphan Register

  35. Orphan and Expired-orphan registers updated.

  36. Added Zokinvy to Orphan register.

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

  38. Added FILSUVEZ to Orphan Register

  39. Added Kymriah & Yescarta to Orphan Register

  40. XENPOZYME added to Orphan Register.

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

  42. Added OXBRYTA to Orphan Register

  43. Updated 'Authorised orphan indication' for Ngenla.

  44. Orphan indication updated for Polivy.

  45. Added ABECMA to the Orphan register.

  46. Scemblix added to Orphan register

  47. Added Kimmtrak to the Orphan Register

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

  49. Ngenla added to Orphan Register

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

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

  52. Updated entries on the 'Orphan Register'

  53. Plenadren moved to expired list

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

  55. QINLOCK added to the Orphan Register

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

  57. Imbruvica removed from register

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

  59. 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.

  60. Added Koselugo to the Orphan register

  61. Mozobil moved to expired orphan registration page

  62. Updated Orphan register with Palynziq and OXERVATE

  63. Newly added entry to Orphan Register - Hepcludex

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

  65. Added Adakveo to the Orphan Register list.

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

  67. 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).

  68. Added Inrebic to the Orphan Register list.

  69. Added Pemazyre to the Orphan Register

  70. 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.

  71. Added information on orphan conditions to the register entries

  72. First published.