AMT prepares for submission of Marketing Authorization Dossier for AMT-011 (Glybera(R))
- Category: DNA RNA and Cells
- Published on Sunday, 15 June 2008 02:00
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Amsterdam Molecular Therapeutics announced today that it has concluded enrollment and treated the last patient in the pivotal trial for it's lead product AMT-011 (Glybera(R)) for Hyperlipoproteinemia, a seriously debilitating and potentially lethal disease
Amsterdam, The Netherlands | June 13, 2008 | Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that it has concluded enrollment and treated the last patient in the pivotal trial for it's lead product AMT-011 (Glybera(R)) for Hyperlipoproteinemia, a seriously debilitating and potentially lethal disease. After recent preparatory meetings with the rapporteur countries Germany and the United Kingdom of the European Medicines Agency (EMEA), AMT expects to file the Marketing Authorization Dossier with EMEA not later than the fourth quarter of 2008.
AMT has developed AMT-011 as a cure for patients with the rare genetic disorder Hyperlipoproteinemia (HPL) type I, also known as LPL-deficiency. These patients have extremely high fat levels in their blood resulting in recurrent and potentially lethal pancreatitis as well as an increased risk of cardiovascular complications and diabetes. Because of a defective gene HPL-patients do not produce an enzyme that normally breaks down fats in the blood. Currently, there is no effective treatment or cure for this seriously debilitating and potentially lethal disease.
AMT-011 is a gene therapy product designed to insert a healthy gene into the patient's body to replace the defective gene. The product is administered by a series of injections into muscle tissue after which the body starts to make the missing enzyme again. The advantage of this therapy is its potential to cure a disease instead of just treating the symptoms. AMT expects to finish the ongoing clinical trial with AMT-011 according to plan.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) 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. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development.
SOURCE: Amsterdam Molecular Therapeutics