1st european appoval for a gene therapy drug

The European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended, last Friday, the authorisation of Glybera (alipogene tiparvovec) for marketing in the European Union. For Fulvio Mavilio, Genethon CSO, that’s “great news for the gene therapy field".

Glybera is a treatment for patients who cannot produce enough of an enzyme that is crucial for breaking down fat. Glybera uses a virus that is injected into the patient to deliver a working copy of a gene for producing lipoprotein lipase (LPL). LPL deficiency is very rare, affecting no more than one or two people in every million.

Learn more about the first european approval for a gene therapy

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Illustration for news: Gene therapy: Généthon starts a clinical trial to test a gene therapy treatment for a rare liver disease, Crigler-Najjar Syndrome

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