Utrecht, The Netherlands – uniQure (Amsterdam, the Netherlands) has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) 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 Company expects final approval from the European Commission within 3 months after the CHMP decision. 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.
As part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPLD and by specially trained doctors to ensure ongoing safety of this novel treatment method. uniQure has also committed to building a patient registry for continued understanding of this devastating, under-researched disease. The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets.
The uniQure team is also developing treatments for a number of other rare diseases, such as acute intermittent porphyria and Sanfilippo B. The potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with uniQure’s proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, the Company is advancing programs in degenerative diseases such as Parkinson’s.