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 EMAwebsite. 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 |
| Alofisel® (darvadstrocel) | Tigenix, S.A.U. | Complex anal fistulas in adults with Crohn’s disease | 23/03/2018 |
| Amglidia® (glibenclamide) | Ammtrek | Neonatal diabetes | 24/05/2018 |
| Crysvita® (burosumab) | Kyowa Kirin Limited | X-linked hypophosphataemia | 19/02/2018 |
Jorveza® (budesonide) | Dr. Falk Pharma GmbH | Eosinophilic oesophagitis | 08/01/2018 |
Lamzede® (velmanase alfa) | Chiesi Farmaceutici S.p.A. | Alpha-mannosidosis | 23/03/2018 |
| Mepsevii® (vestronidase alfametreleptin) | Ultragenyx Germany GmbH | Mucopolysaccharidosis type VII (MPS VII, also known as Sly syndrome) | 23/08/2018 |
| Myalepta® (metreleptin) | Aegerion Pharmaceuticals B.V. | Lipodystrophy | 30/07/2018 |
Mylotarg® (gemtuzumab ozogamicin) | Pfizer Limited | Acute myeloid leukemia | 19/04/2018 |
| Premyvis® (letermovir) | Merck Sharp & Dohme Limited | Cytomegalovirus infection prevention following haematopoietic stem cell transplant | 08/01/2018 |
Rubraca® (rucaparib) | Clovis Oncology UK Limited | High-grade cancers of the ovary, fallopian tubes and peritoneum | 24/05/2018 |
| Teedgsi® (inotersen sodium) | Ionis USA Ltd | Hereditary transthyretin amyloidosis (hATTR) | 06/07/2018 |
| Verkazia® (ciclosporin) | Santen Oy | Keratoconjunctivitis (VKC) in children and adolescents from 4 to 18 years of age. | 06/07/2018 |
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