Thumbs down from EMA for Amsterdam Molecular's Glybera

Dutch human gene therapy firm Amsterdam Molecular Therapeutics (Euronext: AMT) says it has received a negative opinion on its Marketing Authorization Application for Glybera (alipogene tiparvovec) as a potential therapy for the rare genetic disorder lipoprotein lipase deficiency (LPLD). Following a recent meeting with the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), AMT has been notified that, at this time, Glybera is not approvable. The news caused the firm’s shares slump 50%.

Subsequent to a review of the CHMP's letter, AMT believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. The company has therefore decided to ask for a re-examination of the clinical data package.

"From what we know today, despite the disappointment, we believe that there is an indication from the CHMP that Glybera could receive approval and that the current opinion at this time is a reflection of insufficient proof of clinical benefit of Glybera as a result of low patient numbers measured for chylomicron handling for at least 12 months post treatment. The CHMP also indicated that if certain additional data from already treated patients would confirm current results by the end of 2011, an approval may be possible," noted Jorn Aldag, chief executive of AMT. "In the dossier, we provided important data showing Glybera is safe and prevents episodes of pancreatitis, the major clinical complication of LPLD."