December 15, 2025

News from the European Medicines Agency

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The European Medicines Agency is an agency responsible for the scientific evaluation, supervision and safety monitoring of medicines in the European Union. There have been some interesting developments in the past months:

• The Committee for Medicinal Products for Human Use (CHMP) of has given a positive opinion to Waskyra™, a groundbreaking ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency. This positive opinion recommends the marketing authorisation of the gene therapy in the EU and needs to be supported by the European Commission to be fully authorised. Once this is done, the different EU countries would need to consider whether they reimburse/cover the medicine and to what extent in a negotiation with the developer of the therapy. More information here: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-rare-disease-wiskott-aldrich-syndrome
• EMA public webinar on medicine shortages, on November 4, aimed at informing patients, consumers and healthcare professionals about EMA’s role in managing shortages as well as providing information about shortages and sharing best practices on how to help prevent and manage a shortage. More information about the webinar is available here: https://www.ema.europa.eu/en/events/medicine-shortages-ema-public-webinar-putting-patients-first