Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMEA) has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.
Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-080 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.
"We are proud to have received this Orphan Drug Designation for the treatment of Duchenne muscular dystrophy," said Jorn Aldag, Chief Executive Officer of AMT. "This designation is an important next step in the development of a treatment for this progressive and devastating disease."
Duchenne muscular dystrophy (DMD) is a severe disease characterized by progressive muscle degeneration. It affects young children, almost exclusively boys, and leads to paralysis and death in young adulthood. The disease is caused by mutations in the dystrophin gene, thereby blocking the production of functional dystrophin protein, an important structural component within muscle tissue. Currently there is no treatment to prevent the fatal outcome of this disease. DMD affects one in 3,500 males, making it the most prevalent of muscular dystrophies.
AMT is developing a gene therapy product for Duchenne muscluar dystrophy based on technology that results in "skipping" of the defective portion of the dystrophin gene resulting in the formation of functional protein. Positive long-term health effects of this approach have been demonstrated in animals.
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), especially diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with nine products at different stages of development.
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. EMEA, 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.Read full story »