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Amsterdam Molecular Therapeutics Receives Opinion on Glybera(R) Marketing Authorisation Application
Date:6/23/2011

AMSTERDAM, June 24, 2011 /PRNewswire/ --


 

- Dossier Re-Examination Process Initiated

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera® (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), AMT has been notified that, at this time, Glybera is not approvable.

Subsequent to a review of the CHMP's letter, AMT believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. AMT has therefore decided to ask for a re-examination of the clinical data package.

"From what we know today, despite the disappointment, we believe that there is an indication from the CHMP that Glybera could receive approval and that the current opinion at this time is a reflection of insufficient proof of clinical benefit of Glybera as a result of low patient numbers measured for chylomicron handling for at least 12 months post treatment. The CHMP also indicated that if certain additional data from already treated patients would confirm current results by the end of 2011, an approval may be possible," noted Jörn Aldag, CEO of AMT. "In the dossier, we provided important data showing Glybera is safe and prevents episodes of pancreatitis, the major clinical complication of LPLD. We appreciate the CHMP's responsibility for caution on such an advanced therapy, so we will work diligently to generate more information and, we hope, ensure that Glybera will still reach patients."

Next steps

As part of the MAA, AMT presented data to the CHMP on Glybera showing evidence in reducing the risk of pancreatitis. Also, the company demonstrated a clear signal of patients' ability to break down large chylomicron molecules, the accumulation of which, after intake of dietary fat, seems to be responsible for the occurrence of pancreatitis in LPLD patients. AMT is initiating study CT-AMT-011-04 to examine further how already treated patients handle chylomicrons over time. In addition, AMT will continue to collect follow up data on pancreatitis incidence. AMT's available funds will support its existing operations beyond the completion of the re-examination process into 2012.

"Based on communication from EMA, we understand that our technology platform using adeno-associated virus (AAV) vectors is approvable," Mr. Aldag added. "While we pursue the re-examination of Glybera, we will also continue development of other products in our pipeline such as hemophilia B and GDNF gene therapy for Parkinson's disease and Huntington's disease. We will also continue our preparations for registrations in Canada and the US."

For investors, AMT will conduct a conference call at 9:00 AM CET on June 24, 2011. To participate in the conference call, please call one of the following telephone numbers 15 minutes prior to the event, using access code 2788745: +44-(0)20-7136-2054 for the UK; +1-718-247-0881 for the US; and + 31-(0)20-201-5468 for the Netherlands. A pdf version of the presentation is available in the news and investor relations sections of the corporate website. Following the presentation, the lines will be opened for a question and answer session. 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 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. 

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SOURCE Amsterdam Molecular Therapeutics B.V
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