Capricor Therapeutics: FDA grants Rare Pediatric Disease Designation to CAP-1002
Capricor Therapeutics announced that the U.S. FDA has granted Rare Pediatric Disease Designation to CAP-1002, Capricor's development candidate for the treatment of Duchenne muscular dystrophy, a debilitating genetic disorder characterized by progressive weakness and chronic inflammation of skeletal, heart, and respiratory muscles. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted to CAP-1002 by the FDA, covers the broad treatment of DMD. Upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher. In April 2017, Capricor announced positive results from its Phase I/II HOPE clinical trial of CAP-1002 in DMD patients with advanced disease. The company recently met with the FDA to discuss potential product registration strategies for this indication, and Capricor plans to commence a randomized, double-blind, placebo-controlled clinical trial of intravenous, repeat-dose CAP-1002 in boys and young men with Duchenne in the second half of this year, subject to regulatory approval. Capricor expects to report 12-month results from the HOPE Trial in the fourth quarter of 2017.