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FDA Grants RASRx1902 Orphan Drug Status as Potential Therapy for Duchenne Muscular Dystrophy

June 20, 2017

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.

“We are passionate about making an impact in areas of unmet needs like Duchenne muscular dystrophy,” Kathleen Rodgers, RASRx’s co-founder, said in a news release. “This lead program at RASRx takes a mutation-independent approach to halting, and possibly reversing, the degenerative progression of Duchenne. Through this approach, we hope to bring relief to patients with Duchenne, as well as other muscular dystrophies. We have been benefitted immensely from CureDuchenne’s knowledge of the disease and familiarity with the needs of the patients.”

RASRx1902 is an experimental oral drug that causes a demonstrated increase in muscle strength and regeneration as well as decreased muscle inflammation, degeneration and tissue death in preclinical studies using animal models of DMD.

CureDuchenne is an initiative dedicated to research, patient care and innovation to improve and extend the lives of patients living with DMD.

Duchenne is characterized by loss of muscular function, including in the heart and lungs, due to the presence of mutations in the gene encoding the dystrophin protein. Mortality rates are frequently associated with cardiopulmonary failure due to cardiomyopathy.