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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
> 2017 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 |
|
Cablivic® (caplacizumab) |
Ablynx NV | Acquired thrombotic thrombocytopenic purpura (aTTP) | 31/10/2018 |
|
Crysvita® (burosumab) |
Kyowa Kirin Limited | X-linked hypophosphataemia | 19/02/2018 |
|
Jorveza® |
Dr. Falk Pharma GmbH | Eosinophilic oesophagitis | 08/01/2018 |
|
Kymriah® (tisagenlecleucel) |
Novartis Europharm Limited | B-cell acute lymphoblastic leukaemia (ALL), in children and young adults up to 25 years and diffuse large B-cell lymphoma (DLBCL) in adults | 22/08/2018 |
|
Lamzede® |
Chiesi Farmaceutici S.p.A. | Alpha-mannosidosis | 23/03/2018 |
|
Luxturna® (voretigene neparvovec) |
Spark Therapeutics Ireland Ltd | Adults and children with loss of vision due to inherited retinal dystrophy | 22/11/2018 |
|
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® |
Pfizer Limited | Acute myeloid leukemia | 19/04/2018 |
|
Namuscla® (mexiletine hcl) |
Lupin Europe GmbH | Non-dystrophic myotonic disorders | 18/12/2018 |
|
Onpattro® (patisiran) |
Alnylam Netherlands B.V. | Hereditary transthyretin-mediated amyloidosis (hATTR) | 26/08/2018 |
|
Premyvis® (letermovir) |
Merck Sharp & Dohme Limited |
Cytomegalovirus infection prevention following haematopoietic stem cell transplant | 08/01/2018 |
|
Rubraca® |
Clovis Oncology UK Limited | High-grade cancers of the ovary, fallopian tubes and peritoneum |
24/05/2018
|
|
Symkevi® (tezacaftor/ivacaftor) |
Vertex Pharmaceuticals (Europe) Ltd. | Cystic fibrosis in patients aged 12 years and above | 31/10/2018 |
|
Tegsedi® (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 |
|
Vyxeos® |
Jazz Pharmaceuticals Ireland Limited | Adults with newly diagnosed acute myeloid leukaemia | 22/08/2018 |
|
Yescarta® |
Kite Pharma EU B.V. | Diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL) | 23/08/2018 |
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The voice of rare disease patients in Europe
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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