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FDA Gives Muscular Dystrophy Therapy Orphan Drug Status

DNA fragment (Wikimedia Commons)

(Wikimedia Commons)

Amsterdam Molecular Therapeutics (AMT), a biotechnology company in the Netherlands doing work in human gene therapy, said today that the U.S. Food and Drug Administration (FDA) has designated its gene therapy for Duchenne muscular dystrophy (DMD) as an orphan drug. In October 2009, a committee the European Medicines Agency also granted the therapy, known as AMT-080, orphan designation for the the European Union.

AMT says the therapy has shown effective in studies of a preclinical model of DMD. These proof of concept studies demonstrated that the technology resulted in functional dystrophin synthesis in both the heart and skeletal muscles, leading to the prevention of muscular dystrophy. Dystrophin is a protein that helps keep muscle cells intact.

The data are strengthened, says AMT, 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 clinical trial is scheduled to start by the end of 2012.

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. Symptoms include generalized weakness and muscle wasting first affecting the muscles of the hips, pelvic area, thighs and shoulders. DMD eventually affects all voluntary muscles, and the heart and breathing muscles.

The FDA’s orphan drug designation encourages research and development of new therapies for diseases that affect fewer than 200,000 U.S. residents. The company says that with the orphan drug designation, 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.

Related: FDA Grants Orphan Status for Neuromuscular Drug

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