AMSTERDAM, The Netherlands, August 18, 2010 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera(R), a gene therapy product for lipoprotein lipase deficiency (LPLD), remains on track for a regulatory decision by mid-2011.
AMT has had two meetings with the Committee for Advanced Therapy Medicinal Products (CAT) at the EMA for clarification about the Day 120 questions. These meetings have enabled AMT to finalise its strategy for responding to these questions in a timely and effective manner.
The outcome of the meetings suggests that AMT will not be required to conduct more clinical trials with additional new patients to be treated at this time. The responses to the questions will be based in part on additional data and analyses from patients previously treated with Glybera(R). This will include new data available from the last clinical trial (CT-AMT-011-02) and its one year extension.
"We have developed a clear response strategy which, if executed with no unforeseen adverse events or delays, should allow us to remain on track for an EMA decision in the middle of 2011," noted Jorn Aldag, CEO of Amsterdam Molecular Therapeutics. "The route to registration for an innovative product such as Glybera(R) is not only very important for AMT, but it also gives hope to thousands of patients suffering from rare diseases. Gene therapy carries the promise to be able to cure a range of diseases thought to be caused by a single gene. AMT will exert every effort to succeed and reach the market with this unique product."
About the Disease
LPLD is a seriously debilitating 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. This can result not only in potentially lethal pancreatitis, but also in difficult-to-treat diabetes, and is associated with significant morbidity and mortality.
About Amsterdam Molecular Therapeutics
AMT is a leader in the development of human gene based therapies. 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 what is probably the first stable and scalable AAV production platform. This proprietary platform can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPLD, Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and, Parkinson's Disease at different stages of research or development. AMT was founded in 1998 and is based in Amsterdam.
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 Amsterdam Molecular Therapeutics 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, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.
SOURCE Amsterdam Molecular Therapeutics B.V