United States-based Capricor Therapeutics has received the regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational cell therapy for the treatment, CAP-1002 intended to treat Duchenne muscular dystrophy, it was reported yesterday.

The US FDA offers the RMAT designation to regenerative medicine therapies intended to treat a serious condition and for which preliminary clinical evidence indicates a potential to address unmet medical needs for that condition. The RMAT designation makes therapies eligible for the same actions to accelerate the development and review of a marketing application that is available to drugs that receive breakthrough therapy designation including increased meeting opportunities, early interactions to discuss any potential surrogate or intermediate endpoints and the potential to support accelerated approval.

To receive the RMAT designation, the company submitted data from an earlier trial, the HOPE-Duchenne Trial, which reported significant and sustained improvements in skeletal muscle function and cardiac structure in boys and young men in advanced stages of Duchenne muscular dystrophy following a single dose of intracoronary CAP-1002. Capricor is launching a potential registration trial, the HOPE-2 Trial, to test the potential benefit of CAP-1002 as a multi-dose therapy delivered intravenously.