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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
See a list of orphan medicines currently authorised in the EU.
|
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 |
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The voice of rare disease patients in Europe
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
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