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Gene therapy back on track
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Gene therapy back on track

23.07.2012 - A ray of hope for Dutch gene therapy specialist uniQure B.V.: EMA's CHMP
recommended approval of its product Glybera to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks.

EMA said Glybera (alipogene tiparvovec) is the first gene therapy drug to receive a positive CHMP recommendation. The news is a relief for Amsterdam-based uniQure, after having received three negative opinions before. CHMP said it reversed its prior negative opinions following a draft positive opinion in June from the Committee for Advanced Therapies (CAT),which conducted additional analyses of the data for the adeno-associated virus (AAV) vector encoding LPL gene. CHMP said Glybera should be approved under EMA's exceptional circumstances clause, and that the company be required to establish a patient registry to monitor outcomes. uniQure expects a final decision from the European Commission within three months.

UniQure's predecessor, Amsterdam Molecular Therapeutics Holding N.V., first applied for European approval of the gene therapy in December 2009 for all patients with LPL deficiency. CHMP subsequently issued three negative opinions for Glybera, the most recent of which came in April following arequest by the EC to consider the product in a restricted patient population with severe or multiple pancreatitis attacks. Amsterdam Molecular dissolved into uniQure this February.

LPL disease is a very rare, inherited disease which affects 1-2 people per million. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain andpancreatitis. "After today's positive recommendation, Glybera is poised tobecome the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need", Jörn Aldag, CEO of uniQure, said.

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