2019 marketing authorisations


Medicinal Product

Marketing Authorisation Holder

Therapeutic Indication

Date of Marketing Authorisation

BioMarin International Limited Phenylketonuria
Akcea Therapeutics Ireland Limited Hyperlipoproteinemia Type I 03/05/2019
(Autologous CD34+ cells encoding βA-T87Q-globin gene)
bluebird bio (Netherlands) B.V. beta-Thalassemia 29/05/2019
GW Pharma (International) B.V. Lennox-Gastaut and
Dravet syndromes
Astellas Pharma Europe B.V. Acute myeloid leukaemia (AML) 24/10/2019


Marketing authorisations since 2000

Page created: 06/03/2017
Page last updated: 06/03/2020
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseasesRare Disease International Bringing together patients, families and experts to share experiences in a moderated multi-language forum. RareConnect 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.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day 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 initiativeEuropean Conference on Rare Diseases