FDA Awards Capricor’s Duchenne Therapy CAP-1002 Rare Pediatric Disease Designation

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Capricor CAP-1002

The U.S. Food and Drug Administration (FDA) has granted Capricor Therapeutics rare pediatric disease designation for CAP-1002, its candidate therapy to treat  Duchenne muscular dystrophy (DMD).

This latest approval, along with the FDA’s recent approval of orphan drug status to CAP-1002, “underscores the urgent need for treatment options for this devastating rare disease as well as provides Capricor with certain incentives for their development,” the company’s president and CEO, Linda Marbán, said in a press release.

“We believe CAP-1002 has the potential to provide meaningful clinical benefit to patients,” she said. “We look forward to commencing our next clinical trial in DMD, for which design will be relevant to the overall registration plan for this candidate.”


DMD is a genetic condition in which muscle tissue is compromised by the absence of the protein dystrophin. The lack of dystrophin in the heart muscle causes it to become enlarged, thick and rigid; this is actually the leading cause of death in DMD patients. DMD occurs in about one out of 3,000 male births.

CAP-1002 is made of partially compatible cardiac stem cells, or cardiosphere-derived cells (CDCs) — a type of progenitor cell capable of modifying or regulating one or more immune functions. These CDCs promote growth of mature heart cells and vessels, and modulate the immune system to promote heart repair. CAP-1002 effectively improves heart function and exercise capacity while preventing oxidative stress, scarring and inflammation, preclinical studies have found.

According to Capricor, CDCs have been the subject of more than 100 peer-reviewed scientific publications and have been administered to roughly 140 human subjects across several clinical trials.

Earlier this year, Capricor announced positive results from its Phase 1/2 HOPE clinical trial (NCT02485938) of CAP-1002 in DMD patients with advanced disease. The pharmaceutical expects to report one-year results in the fourth quarter of 2017 from that trial, which is funded in part by the California Institute for Regenerative Medicine.

The company, based in Beverly Hills, California, will report one-year results in late 2017. It plans to initiate another trial this year, evaluating CAP-1002 in boys and young men with DMD, following regulatory approval.