- 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.
|
Elzonris® (tagraxofusp) |
Stemline Therapeutics B.V. |
Adults with blastic plasmacytoid dendritic cell neoplasm (BPDCN) |
07/01/2021 |
|
Inrebic® (fedratinib) |
Celgene Europe BV | Adults with myelofibrosis (a rare form of blood cancer) | 08/02/2021 |
|
Lumoxiti® (moxetumomab pasudotox) |
AstraZeneca AB | Adults with hairy cell leukaemia, a cancer of the white blood cells | 08/02/2021 |
|
Sogroya® (somapacitan) |
Novo Nordisk A/S |
Growth hormone deficiency |
31/03/2021 |
|
Enspryng® (satralizumab) |
Roche Registration GmbH |
Neuromyelitis Optica Spectrum disorders (NMOSD) |
24/06/2021 |
|
Bylvay® (odevixibat) |
Albireo | +6 months with progressive familial intrahepatic cholestasis (PFIC) | 16/07/2021 |
|
Skysona® (elivaldogene autotemcel) |
bluebird bio (Netherlands) B.V. | Under 18 years of age with early cerebral adrenoleukodystrophy (CALD) | 16/07/2021 |
|
Imcivree® (setmelanotide) |
Rhythm Pharmaceuticals Limited | +6 years who have pro-opiomelanocortin (POMC) deficiency or leptin receptor (LEPR) deficiency | 16/07/2021 |
|
Abecma® (idecabtagene vicleucel) |
Celgene Europe BV | Adults with multiple myeloma (a cancer of the bone marrow) | 18/08/2021 |
|
Voxzogo® (vosoritide) |
BioMarin International Limited | Achondroplasia in patients aged +2 years | 26/08/2021 |
|
Minjuvi® (tafasitamab) |
Incyte Biosciences Distribution B.V. | Adults with diffuse large B-cell lymphoma (DLBCL) | 26/08/2021 |
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