FDA grants RASRx1902 orphan drug status as potential therapy for Duchenne muscular dystrophy | DuchenneXchange

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FDA grants RASRx1902 orphan drug status as potential therapy for Duchenne muscular dystrophy


The U.S. Food and Drug Administration has granted RASRx1902 orphan drug status to treat Duchenne muscular dystrophy (DMD), says its developer, RASRx of Newport Beach, California.

The Orphan Drug status is given to therapies intended to prevent and treat rare life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people, and for which there are no, or only unsatisfactory, prevention and treatment options. It also awards exclusivity and protects such products from competition for a specified time period once they reach the market.

Based on this designation, RASRx will receive financial incentives aimed at advancing the development of RASRx1902. The will join CureDuchenne in investigating RASRx1902 in trials of DMD patients. CureDuchenne is speeding up development of a clinical trial research program with RASRx1902 along with a research grant provided by the U.S. Department of Defense.

 

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