Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, has received a request to provide additional information to the Committee for Human Medicinal Products (CHMP) on Glybera (alipogene tiparvovec) by March 8, 2012.
The CHMP request follows the announcement on January 30, 2012 that the European Commission Standing Committee was requesting additional information from the CHMP. The CHMP has indicated that a final decision on Glybera could be made by May 2012.
Glybera is a gene therapy for patients with the genetic disorder LPLD, a very rare inherited condition that is associated with increased levels of chylomicrons. These particles carry certain types of fat in the blood, which because they are not removed from the body can cause recurrent bouts of pancreatitis. There is no treatment currently available for these patients.
On October 23, 2011, AMT received a non-approvable opinion from the CHMP (following a re-examination of the marketing approval dossier originally rejected in June 2011) despite a recommendation to approve Glybera by the Committee for Advanced Therapies (CAT). The CAT is an expert body implemented by the European
Commission to prepare and advise the CHMP on decisions regarding advanced therapeutics, in particular gene and cell therapies.
AMT’s proposed acquisition of assets by uniQure BV announced on February 16, 2012, was developed on the basis that Glybera is not approvable. This transaction will proceed as planned. In the event that Glybera could be commercialized in Europe, uniQure will evaluate how achieving this important milestone could be realized most efficiently.
AMT has developed Glybera as a treatment for patients with the genetic disorder lipoprotein lipase deficiency.
LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating and life threatening clinical complication of LPLD. Glybera has orphan drug status in the EU and US.