Company hopes new data from patients already treated using Glybera will be sufficient to reverse CHMP opinion.
EMA’s CHMP has informed Amsterdam Molecular Therapeutics (AMT) that its lipoprotein lipase deficiency (LPLD) candidate, Glybera, can’t be approved on the basis of existing clinical data. The firm says that communication from the committee does, however, suggest that it may be able to achieve approval by generating additional data from existing patients. As a result the firm has asked for a re-examination of the clinical data package.
“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,” notes Jorn Aldag, CEO.
“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.”
Glybera is a gene therapy product that induces functional lipoprotein activity in patients with LPLD. The disease causes highly decreased or complete lack of functional LPL activity, resulting in the accumulation of chylomicrons that can lead to severe pancreatitis.
The Glybera dossier presented to the European agency included evidence that the drug reduces the risk of pancreatitis in LPLD patients and that treatment improves the breakdown of large chylomicron molecules. The firm is in addition starting a study, designated CT-AMT-011-04, to further examine how already treated patients handle chylomicrons over time. Additional follow-up data will be collected on pancreatitis incidence.