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Amsterdam Molecular Therapeutics (AMT) (RHP.DE) Receives Opinion on Re-examination of Glybera(R) Marketing Authorisation Application


10/21/2011 11:49:40 AM

AMSTERDAM, The Netherlands, October 21, 2011 /PRNewswire/ --

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has maintained its earlier opinion that Glybera (alipogene tiparvovec) is not approvable at this time. The decision was made contrary to the positive recommendation of CHMP Rapporteurs, the Scientific Advisory Group (SAG), an expert panel specifically selected to evaluate clinical results and the science of the product, and the Committee for Advanced Therapies (CAT), which provides guidance on advanced therapeutics such as gene and cell therapy. These expert groups after extensive review and analysis of the data advised the CHMP that Glybera should be approved now under exceptional circumstances. The CHMP Rapporteurs, SAG and the CAT concluded that data from three Glybera clinical trials demonstrated meaningful evidence of clinical efficacy, without any major safety concerns. However, the CHMP is not bound to follow this advice. While AMT carefully reviews the grounds of the CHMP refusal, the Company will focus on strategic advancement of other promising projects in its development pipeline.

"The decision from the CHMP is disappointing particularly because a treatment will now not be available to European patients who suffer from LPLD for whom Glybera is the only therapeutic option. We are encouraged by the validation provided by the CHMP Rapporteurs, Scientific Advisory Group and the Committee for Advanced Therapies regarding our gene therapy platform, indicating that safety is not an issue," said Jörn Aldag, CEO of AMT. "We are evaluating thoroughly and expediently the next steps available to the Company and will provide an update on the future developments of our validated gene therapy pipeline."

On Tuesday October 25, 2011 at 10:00 AM CET, AMT will provide an update on the company and its product pipeline to investors on a conference call. To participate in the conference call, please call one of the following telephone numbers 15 minutes prior to the event, using access code 6749807: +31(0)20-721-9158 for the Netherlands and +44(0)20-3450-9987 for the UK. Following the presentation, the lines will be opened for questions. A replay of the call will be available following the event.

About Glybera®

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. In addition to Glybera, AMT has a product pipeline of several gene therapy products in development for hemophilia B, Duchenne muscular dystrophy, 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.

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.


SOURCE Amsterdam Molecular Therapeutics (AMT) B.V



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