Decision
Orphan register
Updated 12 December 2025
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| Orphan registered products are listed in alphabetical order by trade name on the downloadable or browser view CSV file below. The file 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. |
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| Product name |
| ABECMA |
| Adzynma |
| AGAMREE |
| Akantior |
| ALPROLIX |
| ALYFTREK |
| AMGLIDIA |
| ARIKAYCE liposomal |
| Artesunate Amivas |
| ASPAVELI |
| Aucatzyl |
| Avacopan Vifor |
| Avacopan Vifor |
| AYVAKYT |
| AYVAKYT |
| Besponsa |
| BLINCYTO |
| Brineura |
| Bylvay |
| Cablivi |
| Carvykti |
| Casgevy |
| Casgevy |
| Cerdelga |
| Chenodeoxycholic acid Leadiant |
| Coagadex |
| Cresemba |
| Cresemba |
| CRYSVITA |
| Cystadrops |
| Darzalex |
| Darzalex |
| Daurismo |
| Dovprela |
| Duvyzat |
| Ebvallo 2.8 x 10^7 - 7.3 x 10^7 cells/mL dispersion for injection |
| Ekterly |
| ELZONRIS |
| Enspryng |
| Epidyolex |
| Epidyolex |
| Epidyolex |
| Ezmekly |
| FABHALTA |
| FABHALTA???? |
| Filspari |
| FILSUVEZ |
| FINLEE |
| Fintepla |
| Fintepla |
| Galafold |
| Gilteritinib Astellas 40mg film-coated tablets |
| Givlaari |
| Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion |
| Holoclar |
| Hyftor |
| Idefirix |
| IDELVION |
| Imcivree |
| Imcivree |
| IMDYLLTRA 1 milligram and 10mg powder for concentrate and solution for infusion |
| Inrebic |
| IQIRVO |
| Isturisa |
| Ixazomib |
| Joenja |
| Jorveza |
| Kaftrio |
| KIMMTRAK |
| Kinpeygo |
| Koselugo |
| Kymriah |
| Kymriah |
| Kymriah |
| Lamzede |
| Ledaga |
| Libmeldy |
| LIVMARLI |
| LIVTENCITY |
| LIVTENCITY |
| Loargys 5 mg/ml solution for injection/infusion |
| Lonapegsomatropin Ascendis Pharma |
| LUNSUMIO |
| Lutathera |
| Luxturna |
| Luxturna |
| Mepsevil |
| Minjuvi |
| Myalepta |
| Myalepta |
| Myalepta |
| Myalepta |
| Mylotarg |
| Namuscla |
| Natpar |
| Nexviadyme 100 mg powder for concentrate for solution for infusion |
| Ngenla |
| Ocaliva |
| Onivyde pegylated liposomal |
| Onpattro |
| Opfolda |
| OXBRYTA |
| Oxlumo |
| Pemazyre |
| Polivy |
| Pombiliti 105 mg powder for concentrate for solution for infusion |
| Poteligeo |
| PREVYMIS |
| PYRUKYND |
| Qalsody |
| Qarziba |
| QINLOCK |
| Ravicti |
| Ravicti |
| Ravicti |
| Ravicti |
| Ravicti |
| Ravicti |
| Reblozyl |
| REZUROCK |
| Rezzayo |
| Rydapt |
| Rydapt |
| Rystiggo 140 mg/ml solution for injection |
| Scemblix |
| Skyclarys 50 mg hard capsules |
| Sogroya |
| Soliris |
| Soliris |
| SomaKit TOC |
| Spexotras |
| Spinraza |
| Strimvelis |
| Symkevi |
| TAKHZYRO |
| Tecartus |
| Tecartus |
| Tegsedi |
| TIBSOVO |
| TIBSOVO |
| Trecondi |
| Trepulmix |
| UPSTAZA |
| Vanflyta |
| Verkazia |
| Voraxaze 1000 units powder for solution for injection |
| Vyloy |
| Vyndaqel |
| Vyvgart 20 mg/ml Concentrate for solution for infusion |
| Vyvgart 1000 mg solution for injection) |
| Vyvgart 1000 mg solution for injection in pre-filled syringe |
| Vyxeos liposomal |
| Wainzua |
| Waylivra |
| Winrevair |
| XENPOZYME |
| Xermelo |
| Yescarta |
| Yescarta |
| Yescarta |
| Yorvipath 168 micrograms/0.56 mL solution for injection in pre filled pen |
| Yorvipath 294 micrograms/0.98 mL solution for injection in pre filled pen |
| Yorvipath 420 micrograms/1.4 mL solution for injection in pre filled pen |
| Zejula |
| Zokinvy |
| Zolgensma |
| Ztalmy 50 mg/ml Oral suspension |