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First Gene Therapy in Western World Receives Positive Opinion in Europe from CHMP
Date:7/20/2012

AMSTERDAM, The Netherlands, July 20, 2012 /PRNewswire/ --

  • First gene therapy in the Western world to reach important regulatory approval milestone, culminating 40 years of research
  • First therapy for LPL deficient patients, a severe disease with no alternative treatment
  • Validates uniQure's unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale
  • Heralds new phase in uniQure's development, including potential revenues from sales and partnerships
  • Technology platform can now be leveraged to find solutions for many more severe genetic and other disorders

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

The European Commission (EC) generally follows the recommendations of the CHMP. "We expect final approval from the EC within 3 months after the CHMP decision," says Jörn Aldag, CEO of uniQure.  "After today's positive recommendation, Glybera is poised to become 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." Marketing authorization covers all 27 European Union member states.

Mr. Aldag continued: "Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care. Now, for the first time, a treatment exi
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SOURCE uniQure B.V.
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