Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced that it intends to incorporate additional data into the Marketing Authorization Application (MAA) for Glybera for Lipoprotein Lipase Deficiency (LPLD), a seriously debilitating and potentially lethal disease.
The data obtained so far from two phase-II clinical studies (22 patients in total) demonstrate that Glybera is well-tolerated (no drug-related severe adverse events and no dose-limiting toxicity). Positive effects were shown on fat (triglyceride) metabolism, resulting in an almost complete disappearance of pancreatitis during both the immediate 12-week study observation period and the long-term follow-up (up to three years post-injection), as well as the disappearance or reduction of fat accumulations in skin or retina. All patients reported gain of energy and in the two patients that had diabetes, a reduction of insulin resistance was observed, leading to a reduction of their diabetic medication.
In consultation with the Rapporteur countries, Germany and the United Kingdom, of the European Medicines Agency (EMEA) and Health Canada, AMT has decided to expand the clinical experience and to augment the MAA with an additional 16-patient study that will investigate the broader impact of this gene therapy on additional aspects of lipid metabolism. Preparations for this study to be conducted in Canada will start immediately. Submission of the MAA to the EMEA is foreseen in the second half of 2009. In line with the guidance given in the half year financial report, the Company's cash position allows the completion of this study to be done with existing financial resources.
AMT has developed Glybera as a cure for patients with the rare genetic disorder, LPLD. Because of a defective gene, LPLD patients do not produce an enzyme that normally breaks down fats in the blood. 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. Currently, there exists no effective treatment or cure for this serious disease.
Glybera is a gene therapy, which inserts into muscle tissue a healthy gene that restores the enzyme (lipoprotein lipase) activity required to process fat. The advantage of this therapy is its potential to cure a disease instead of just treating the symptoms.
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.