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Glybera first gene therapy to gain EMA approval

By News team

Alipogene tiparvovec has become the first gene therapy to be recommended for approval by the European Medicines Agency.

The drug, branded Glybera by UniQure, is a solution for injection used for treating adults with lipoprotein lipase (LPL) deficiency, a genetic disorder that affects one or two people per million.

Current treatment for LPL deficiency involves restricting dietary fat to less than 20 per cent of daily calorie intake. Alipogene tiparvovec enables LPL production by delivering working copies of the gene to the muscle cells.

After rejecting two previous marketing authorisation applications for the drug, the EMA’s Committee for Medicinal Products for Human Use re-evaluated it and last week gave a positive opinion for its use in the treatment of patients with severe or multiple pancreatitis attacks, and with the requirement that patient outcomes data are shared with the EMA.

Rachel Airley, senior pharmacy lecturer at the University of Huddersfield, said Glybera’s approval is "an exciting and significant development in medicines research".

Dr Airley, who is a member of the Royal Pharmaceutical Society’s English Pharmacy Board, explained: "The discovery and development of genes as medicines has been a difficult process." Development of the technology might lead to treatments for a variety of diseases caused by faulty gene expression, including cystic fibrosis, she suggested.

The CHMP’s opinion on Glybera will now be sent to the European Commission for the adoption of a decision.

Citation: The Pharmaceutical Journal URI: 11104528

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