Amsterdam Molecular Therapeutics (AMT), a leader in the field of human gene therapy, announced that the Marketing Authorisation Application (MAA) for Glybera remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera, 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. 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 Jörn Aldag, CEO of Amsterdam Molecular Therapeutics. “The route to registration for an innovative product such as Glybera 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.”

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.