Duchenne Muscular Therapy Designation Granted RMAT Designation


The U.S. FDA has granted Capricor Therapeutics RMAT designation for its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, CAP-1002.

The U.S. Food and Drug Administration (FDA) has granted Capricor Therapeutics Regenerative Medicine Advanced Therapy (RMAT) designation for its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD), CAP-1002.

RMAT designation is granted to regenerative medicine therapies being developed to treat a serious condition where preliminary clinical evidence proves a potential to address an unmet need.

Data from the HOPE (Halt cardiomyopathy progression in Duchenne) trial was reported in April and exhibited the drug was safe and well-tolerated in patients. The impending HOPE-2 trial plans to enroll an estimated 84 participants in the advanced stages of DMD and will evaluate the potential clinical benefit of CAP-1002 as a multi-dose therapy administered intravenously.

“The RMAT designation is recognition by the FDA of the potential of CAP-1002 and the importance of bringing this therapy to market to serve the unmet needs of boys and young men who have lost the ability to walk because of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Capricor president and chief executive officer in a press release. “CAP-1002 is one of the few therapies currently in development to help non-ambulant patients with Duchenne muscular dystrophy, and it’s important that we move forward into the next phase of clinical development to potentially help them maintain what function they have in their arms and hands.”

A lack of functional dystrophin, the protein intended to keep muscle cells intact, is typically the primary cause of DMD. Patients with the muscle disorder experience symptoms in early childhood, and can lose the ability to walk by age 10. As the disease continues to progress, these primarily male patients are susceptible to life-threatening heart and lung complications.

CAP-1002 is made primarily from a unique population of cells that contain cardiac progenitor cells, or allogeneic cardiosphere-derived cells (CDCs), and has demonstrated the ability to exert potent immunomodulatory activity and stimulate cellular regeneration.

In July, the company was granted Rare Pediatric Disease designation for the potential treatment option, giving it the potential to receive a priority review voucher and fast-track a potential future therapy, should CAP-1002 be approved. Shortly thereafter, the company held a successful meeting with the FDA, in which the regulatory authority approved of plans to continue developing the drug.

Additionally, Capricor has already received orphan drug designation for CAP-1002, giving the company 7-year market exclusivity upon approval.

For more from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter.

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