AMT receives orphan drug designation from the U.S. Food and Drug Administration Duchenne Muscular Dystrophy gene therapy

Amsterdam Molecular Therapeutics (AMT) Holding N.V. (Euronext: AMT), a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has designated AMT-080, a gene therapy for Duchenne muscular dystrophy (DMD) as an orphan drug. In October 2009, the Committee for Orphan Medical Products of the European Medicines Agency granted AMT-080 orphan designation for the same indication in the European Union.
AMT has shown efficacy in studies of a preclinical model of DMD. These proof of concept studies demonstrated that AMT’s technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, leading to the prevention of muscular dystrophy. These data are strengthened by a study in which this gene therapy approach was shown to successfully restore dystrophin activity in diseased human muscle cells obtained from biopsies of DMD patients. A Phase I/II clinical trial is scheduled to start by the end of 2012.
AMT has received an Innovation Credit of up to € 4 million from the Dutch government to support the development of AMT’s gene therapy treatment for Duchenne Muscular Dystrophy (DMD). The credit is granted by SenterNovem, an agency of the Dutch Ministry of Economic affairs.
“It is exceptional that we have been able to reveal the promise of this therapy to the FDA in this early stage of the development. We believe our proven adenoassociated viral vector technology used in all our gene therapy products provides a distinct advantage. AMT has successfully conducted three clinical trials with its lead product Glybera that employs this technology, confirming that AAV-based delivery technology is safe and efficacious,” noted Jörn Aldag, CEO of Amsterdam Molecular Therapeutics.
The FDA's orphan drug designation is intended to encourage research and development of new therapies for diseases that affect fewer than 200,000 U.S. residents. As a designated orphan drug, AMT-080 is eligible for tax credits based on its clinical development costs, as well as assistance from the FDA in guiding the drug through the regulatory approval process. The designation provides the opportunity for AMT-080 to obtain market exclusivity for seven years from the drug's marketing approval date.
--About Duchenne Muscular Dystrophy
DMD is a severe disease characterized by progressive muscle degeneration. It affects young children, almost exclusively boys, and leads to progressive paralysis and death in young adulthood. The disease is caused by mutations in the dystrophin gene, as a result of which the production of functional dystrophin protein, an important structural component within muscle tissue, is blocked. Currently, there is no treatment to prevent the fatal outcome of this disease. DMD affects one in 3,500 males, making it one of the most prevalent of muscular dystrophies.