The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended the authorisation of Glybera (alipogene tiparvovec) for marketing in the European Union. It is intended to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks, despite dietary fat restrictions. LPL deficiency is an ultra-rare inherited disorder estimated to affect no more than 1-2 people per million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats.
Glybera is the first gene-therapy medicine to be recommended for authorisation in the European Union. Gene therapy medicines have the potential to cure genetic disorders by replacing a defective gene with a working copy, thus helping the body to recover functionality. The CHMP’s opinion on Glybera will now be sent to the European Commission for the adoption of a marketing authorisation. For more information, please visit EMA’s site.
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