First Gene Therapy in the West Just Around the Corner

By July 25, 2012Pharma Beat

The European Medicine’s Agency (EMA) has recommended approval of UniQure’s Glybera (alipogene tiparvovec). The European Commission will make the final decision on approval in 3 months, but usually follows the recommendations of the EMA. If Glybera is approved it would be the first gene therapy available in a western country. UniQure has also begun preparations for meetings with the United States Food and Drug Administration.

Glybera treats the rare disorder lipoprotein lipase deficiency (LPLD). Patients with LPLD are unable to produce lipoprotein lipase (LPL), an enzyme which breaks down fat, due to a genetic mutation. As a result fat particles build up in the blood causing abdominal pain and potentially life-threatening pancreatitis. Currently the only treatment is a fat-restricted diet. Glybera is a viral vector containing a healthy copy of the gene responsible for the production of LPL. It is injected into a patient’s muscle, and allows them to produce 5-10% of the LPL a healthy individual would, reducing the occurrence of pancreatitis.

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