Posted by Michael Wonder on 19 Jul 2017
Capricor receives rare paediatric disease designation from FDA for CAP-1002 for patients with Duchenne muscular dystrophy
18 July 2017 - Designation covers broad treatment of Duchenne muscular dystrophy.
Capricor Therapeutics today announced that the U.S. FDA has granted rare paediatric disease designation to CAP-1002, Capricor's development candidate for the treatment of Duchenne muscular dystrophy (DMD), a debilitating genetic disorder characterised by progressive weakness and chronic inflammation of skeletal, heart, and respiratory muscles.
The rare paediatric 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.
