A Duchenne Muscular Dystrophy drug in stage clinical development by GlaxoSmithKline (NYSE:GSK) is now recognized by the Food and Drug Administration as a “breakthrough therapy,” a designation intended to speed regulatory review of treatments for serious or life-threatening conditions.
GSK said that it was notified on Wednesday of the designation for its drug candidate drisapersen. The drug is already classified as an orphan drug in Europe and the United States, a status reserved for drugs that treat rare medical conditions. Orphan drug status comes with tax advantages and marketing exclusivity, if the drug secures approval.
Duchenne Muscular Dystrophy, or DMD, is a childhood neuromuscular disease that affects an estimated 1 in 3,500 live male births. The rare disease is caused by mutations in the dystrophin gene. Patients who have DMD progressively lose muscle strength due to the absence or defect of the dystrophin protein. They are often wheelchair bound by the age of 12. The disease also affects the lungs and the heart and most patients die in early adulthood due to respiratory and cardiac failure.
London-based GSK operates its U.S. headquarters in Research Triangle Park. GSK is developing drisapersen under an exclusive, worldwide license from Dutch company Prosensa.
