Amsterdam Molecular Therapeutics Receives Request for Further Information From CHMP on Glybera®
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(R) (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.
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SOURCE Amsterdam Molecular Therapeutics (AMT) B.V