The Voice of Rare Disease
Patients in Europe
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
Medicinal Product | Marketing Authorisation Holder | Therapeutic Indication | Date of Marketing Authorisation |
| Palynziq® (pegvaliase) | BioMarin International Limited | Phenylketonuria (PKU) | 03/05/2019 |
| Waylivra® (volanesorsen) | Akcea Therapeutics Ireland Limited | Hyperlipoproteinemia Type I | 03/05/2019 |
| Zynteglo® (Autologous CD34+ cells encoding βA-T87Q-globin gene) | bluebird bio (Netherlands) B.V. | beta-Thalassemia | 29/05/2019 |
| Epidyolex® (Cannabidiol) | GW Pharma (International) B.V. | Lennox-Gastaut and Dravet syndromes | 19/09/2019 |
| Xospata® (Gilteritinib) | Astellas Pharma Europe B.V. | Acute myeloid leukaemia (AML) | 24/10/2019 |
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