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
|
Alprolix®
(eftrenacog alfa) |
Biogen Idec Ltd
|
Haemophilia B
|
12/05/2016
|
Coagadex®
(human coagulation factor X) |
Bio Products Laboratory Limited
|
Hereditary factor X deficiency
|
16/03/2016
|
Darzalex®
(daratumumab) |
Janssen-Cilag International N.V.
|
Multiple myeloma
|
20/05/2016
|
Galafold®
(migalastat) |
Amicus Therapeutics UK Ltd
|
Fabry disease
|
16/05/2016
|
Idelvion®
(albutrepenonacog alfa) |
CSL Behring GmbH
|
Haemophilia B
|
11/05/2016
|
Onyvide®
(irinotecan hydrochloride trihydrate) |
Baxalta Innovations GmbH
|
Metastatic adenocarcinoma of the pancreas
|
14/10/2016
|
Strimvelis®
(autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence) |
GlaxoSmithKline Trading Services Limited
|
Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)
|
26/05/2016
|
Uptravi®
(selexipag)* |
Actelion Registration Ltd
|
Pulmonary arterial hypertension
|
12/05/2016
|
Wakix®
(pitolisant) |
Bioprojet Pharma
|
Narcolepsy
|
31/03/2016
|
Zalmoxis®
(allogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor and the herpes simplex I virus thymidine kinase) | MolMed SpA | Adjunctive treatment in adults who have received a haematopoietic stem cell transplant from a partially matched donor | 18/08/2016 |
*Removed from the Community register of orphan medicinal products upon request of the MAH at the time of marketing authorisation
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