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List of the latest marketing authorisations and orphan medicinal products designations
Detailed information on European orphan medicinal products designation applications is available on the EMA website. A full list of designated and authorised orphan medicinal products in Europe available at: ec.europa.eu.
Learn more about the Orphan designation process in Europe
Recent marketing authorisations
See a list of orphan medicines currently authorised in the EU.
|
Medicinal Product |
Marketing Authorisation Holder |
Therapeutic Indication |
Date of Marketing Authorisation |
|
Isturisa® (osilodrostat) |
Novartis Europharm Limited | Cushing Syndrome | 09/01/2020 |
|
Polivy® (polatuzumab vedotin) |
Roche Registration GmbH | Diffuse large B-cell lymphoma (DLBCL) | 16/01/2020 |
|
Givlaari® (givosiran) |
Alnylam Netherlands B.V. |
Acute hepatic porphyria |
02/03/2020 |
|
Trepulmix® (treprostinil) |
SciPharm Sàrl |
Chronic thromboembolic pulmonary hypertension (CTEPH) |
03/04/2020 |
|
Zolgensma® (onasemnogene abeparvovec) |
AveXis EU Limited |
Spinal muscular atrophy (SMA) |
18/05/2020 |
|
Reblozyl® (luspatercept) |
Celgene Europe B.V. | Beta thalassaemia & Myelodysplastic syndromes | 25/06/2020 |
|
Daurismo® (glasdegib) |
Pfizer Europe MA EEIG | Newly-diagnosed acute myeloid leukaemia (AML) | 26/06/2020 |
|
Pretomanid FGK (pretomanid) |
FGK Representative Service GmbH | Adults with drug-resistant tuberculosis | 31/07/2020 |
|
Hepcludex (bulevirtide) |
MYR GmbH | Chronic (long-term) hepatitis delta virus (HDV) infection in adults | 31/07/2020 |
|
Idefirix (imlifidase) |
Hansa Biopharma AB | Prevent the body from rejecting a newly transplanted kidney | 25/08/2020 |
|
Kaftrio (ivacaftor / tezacaftor / elexacaftor) |
Vertex Pharmaceuticals (Ireland) Limited | Cystic fibrosis | 21/08/2020 |
|
Blenrep (belantamab mafodotin) |
GlaxoSmithKline (Ireland) Limited | Multiple Myeloma | 25/08/2020 |
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The voice of rare disease patients in Europe
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Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative
The international voice of people living with rare diseases
Bringing together patients, families and experts to share experiences in a moderated multi-language forum.
The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.
An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiative
Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiative