EMA confirms Glybera decision

 The EMA’s CHMP has maintained its decision not to recommend a marketing authorisation for the orphan medicine Glybera (alipogene tiparvovec).

The Committee again reviewed the benefit risk of Glybera in lipoprotein lipase deficiency patients with severe or multiple pancreatitis attacks.

But it concluded there was insufficient evidence to show the benefit of the gene-therapy in the restricted group of patients and was unable to recommend marketing authorisation.

The European Commission requested in January 2012 that the EMA review its negative opinion confirmed in October 2011. That followed a request from the applicant Amsterdam Molecular Therapeutics B.V. after the CHMP had originally failed to recommend the marketing of the product in June 2011.

Glybera is a gene-therapy product that uses an adeno-associated viral vector intended to treat adult patients diagnosed with lipoprotein lipase deficiency demonstrating hyperchylomicronaemia or who have a history of acute pancreatitis.

But the CHMP found it difficult to obtain and assess data in this very rare disease.

After considering all of the evidence, it concluded that Glybera reduced pancreatitis attacks in the small number of patients assessed, but the evidence was not sufficiently convincing.

In addition, the Committee decided that the reduced risk of pancreatitis attacks may have been due to other factors, such as lifestyle and diet.