Amsterdam Molecular Therapeutics (AMT) Receives Request for Further Information From CHMP on Glybera(R)
Published: Feb 21, 2012
AMSTERDAM, The Netherlands, February 21, 2012 /PRNewswire/ --
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that it 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 17, 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.
About Amsterdam Molecular Therapeutics
AMT is a world leader in the development of human gene based therapies. AMT has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows AMT to pursue its strategy of focusing on this sector of the industry. AMT was founded in 1998 and is based in Amsterdam. Further information can be found at http://www.amtbiopharma.com.
uniQure BV is a private company created specifically for the transaction with AMT. It is funded by Forbion Capital Partners, an existing investor in AMT. uniQure will act as the new holding company for the gene therapy business currently carried out by AMT.
Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law.
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SOURCE Amsterdam Molecular Therapeutics (AMT) B.V