jueves, 23 de enero de 2020

List of the latest marketing authorisations and orphan medicinal products designations | www.eurordis.org

List of the latest marketing authorisations and orphan medicinal products designations | www.eurordis.org

Eurordis, Rare Diseases Europe

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.

Orphan drug regulatory processLearn more about the Orphan designation process in Europe








Recent marketing authorisations

Medicinal Product
Marketing Authorisation Holder
Therapeutic Indication
Date of Marketing Authorisation
Palynziq®
(pegvaliase)
BioMarin International LimitedPhenylketonuria
(PKU)
03/05/2019
Waylivra®
(volanesorsen)
Akcea Therapeutics Ireland LimitedHyperlipoproteinemia Type I03/05/2019
Zynteglo®
(Autologous CD34+ cells encoding βA-T87Q-globin gene)
bluebird bio (Netherlands) B.V.beta-Thalassemia29/05/2019
Epidyolex®
(Cannabidiol)
GW Pharma (International) B.V.Lennox-Gastaut and
Dravet syndromes
19/09/2019
Xospata®
(Gilteritinib)
Astellas Pharma Europe B.V.Acute myeloid leukaemia (AML)24/10/2019

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