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US FDA & EMA grant orphan drug status to Audentes Therapeutics' AT001 to treat X-linked myotubular myopathy

San Francisco, California
Saturday, August 29, 2015, 14:00 Hrs  [IST]

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted orphan drug designation to biotechnology company Audentes Therapeutics Inc's AT001, an investigational product in development for the treatment of X-linked myotubular myopathy (XLM).

XLMTM is a rare, severe, inherited disorder that affects skeletal muscles from birth. The disease is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin plays an important role in the development, maintenance and function of muscle cells. XLM is characterized by debilitating muscle weakness, respiratory impairment and early mortality and affects approximately 1 in 50,000 newborn males worldwide. There is no available treatment for the condition.

AT001 is a novel product based on adeno-associated virus (AAV) gene therapy technology that is designed to restore the ability of skeletal muscle to produce myotubularin. Administration of AT001 has resulted in improved muscle strength and survival in a murine model of XLM as well as improved muscle strength, respiratory symptoms and survival in a canine model of the disease.

The FDA and EMA Orphan Drug programmes provide orphan designation to drugs and biologics that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States or 5 in 10,000 in the EU). The designation provides sponsors with development and commercial incentives including, seven years of market exclusivity in the US, 10 years of market exclusivity in the EU, consultation by FDA and EMA on clinical study design, and certain fee exemptions and reductions.


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