Amsterdam, The Netherlands – Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the Marketing Authorisation Application (MAA) for Glybera®, AMT’s proprietary lead product, for lipoprotein lipase deficient patients, has been submitted to the European Medicines Agency (EMA, formerly known as EMEA).
AMT has conducted two clinical studies for lipoprotein lipase deficiency (LPLD) in Europe and Canada and long term follow-up from these is ongoing, as is a further study in Canada. In these three studies Glybera® has shown a sizeable decrease in the incidence of pancreatitis, or acute inflammation of the pancreas, the most debilitating complication of LPLD. Moreover, these studies also indicate that Glybera® has an excellent safety profile.
“We are delighted to have submitted the Glybera® dossier to EMA. We believe that AMT will be the first to successfully develop a gene therapy for a disease caused by a genetic defect,”
said Jörn Aldag, Chief Executive Officer of AMT.
“Importantly, Glybera® also validates AMT’s adeno-associated viral (AAV) vector delivery platform, which can be used to deliver other gene therapy products for other indications.”
The MAA for Glybera® is now in the validation stage, and the formal review process is expected to begin later this month. The MAA for Glybera® will be reviewed via the centralised procedure which is the standard route for all advanced therapies.
About the Disease
LPLD is a seriously debilitating, and potentially lethal, orphan disease, for which no approved therapy 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, which in turn can lead to pancreatitis. Recurrent pancreatitis in LPLD patients can result in difficult-to-treat diabetes. LPLD is associated with significant morbidity and mortality.
About Amsterdam Molecular Therapeutics
AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using AAV 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 safe and efficacious proprietary platform offers a unique manufacturing capability which 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, DMD, Acute Intermittent Porphyria and Parkinson’s Disease at different stages of research or development.
Chief Executive Officer
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