glybera

AMSTERDAM, The Netherlands, July 20, 2012

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.

Gene therapy suffered another blow, when Dutch biotech company Amsterdam Molecular Therapeutics, AMT, got another negative opinion for it's lead drug Glybera. The opinion was already a second for Glybera, since the company got first negative opinion in July 2011 and asked for re-examination from EMA.

Interestingly, this time the company got a positive opinion from CAT, Committee of Advanced Therapies, which might indicate that the product was deemed safe and effective, but CHMP, The Committee for Medicinal Products for Human Use, voted to yield a negative decision.