Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, was informed on January 27 after business hours that the European Commission’s Standing Committee of the European Parliament discussed on January 22 the implementation decision not to grant marketing authorization for Glybera (alipogene tiparvovec) as recommended by the Committee for Human Medicinal Products (CHMP).
After the discussion no clear position in favour or against granting a marketing authorization for Glybera was reached. Instead the Standing Committee considered it necessary to request additional information to the CHMP in the European Medicines Agency (EMA). A formal vote by the Standing Committee will be made on review of the additional information. It is currently unclear when a final decision will be reached.
Glybera is a gene therapy for patients with the genetic disorder lipoprotein lipase deficiency (LPLD). Currently no alternative treatment is available for patients deficient of LPL, who are suffering from recurrent pancreatitis.
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.
“We are very encouraged by the outcome of the discussion by the European Parliament’s Standing Committee to request further information from the CHMP. The evaluation of (ultra-) orphan drugs and advanced therapies is novel territory. The ongoing debate shows that the authorities are determined to find the best process of dealing with such innovative treatments and technologies. We will constructively work with the European Commission and the EMA to find the best process going forward, not only for Glybera but also for further advanced therapies on their way to patients,” said Jörn Aldag, CEO of AMT. “While gaining clarity for the process involving Glybera, we will continue to implement our revised business strategy revealed following the last CHMP decision. This involves moving forward in partnering discussions for several of our gene therapy products, including haemophilia B and GDNF.”
AMT’s current business plan, organizational structure and financing have been developed on the basis of the current CHMP opinion for Glybera’s non-approval. It involved a reduction of its workforce by about 50 per cent. In the event that Glybera could be commercialized in Europe, the Company will evaluate how the opportunity arising from passing 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.