This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC's registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

23 Jul 2012

EMA Recommends Approval of Glybera

The Agency has recommended approval of Glybera, the world’s first gene therapy drug.

The European Medicines Agency has recommended approval of Glybera (alipogene tiparvovec), the world’s first gene therapy drug, after rejecting it on three previous occasions.


The drug, manufactured by Dutch biotechnology company uniQure, is a treatment for lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks.


LPL deficiency is an extremely rare genetic disorder estimated to affect only one or two people in a million. Due to a defective gene, patients with this disorder cannot produce enough LPL, an enzyme responsible for breaking down fats.


EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the marketing authorisation under ‘except

Related News