- About EURORDIS
- About Rare Diseases
- Rare Disease Policy
- Orphan Drugs & Treatments
- What is an orphan drug?
- Improve the safety of your medicines
- Compassionate Use
- Role of EURORDIS
- Patient advocates involvement
- Access to orphan drugs
- Health technology assessment
- EURORDIS Round Table of Companies (ERTC)
- List of the latest marketing authorisations and orphan medicinal products designations
- Living with a Rare Disease
- Services & Trainings
- Get involved
- News & Events
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 |
|
Ayvakyt (avapritinib) |
Blueprint Medicines (Netherlands) B.V. | Gastrointestinal stromal tumour (GIST) | 24/09/2020 |
|
Arikayce liposomal (amikacin sulfate) |
Insmed Netherlands B.V. |
Adults with a lung infection caused by Mycobacterium avium complex (MAC) |
27/10/2020 |
|
Adakveo (crizanlizumab) |
Novartis Europharm Limited | Anemia, Sickle Cell | 28/10/2020 |
|
Obiltoxaximab SFL (obiltoxaximab) |
SFL Pharmaceuticals Deutschland GmbH | Anthrax | 18/11/2020 |
|
Oxlumo (lumasiran) |
Alnylam Netherlands B.V. | Hyperoxaluria, Primary | 19/11/2020 |
|
Libmeldy (autologous CD34+ cells encoding ARSA gene) |
Orchard Therapeutics (Netherlands) BV |
Metachromatic leukodystrophy (MLD) |
17/12/2020 |
|
Fintepla® (fenfluramine) |
Zogenix ROI Limited |
Seizures associated with Dravet syndrome |
18/12/2020 |
Find out the latest news from the rare disease community! We comply with the HONcode standard for health trustworthy information
We are grateful for the financial support of the EURORDIS website by:
This website received funding under an operating grant from the European Union’s Health Programme (2014-2020)
The voice of rare disease patients in Europe
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
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