Orphan Drug Designation Granted to Capricor’s Therapy for DMD-Related Cardiomyopathy

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Clinical-stage biotechnology company Capricor Therapeutics, Inc. was recently awarded orphan drug designation for its investigational cell therapy CAP-1002, from the U.S. Food and Drug Administration (FDA). The agency granted the status to the company, which is working on the development of CAP-1002 to treat cardiomyopathy related to Duchenne muscular dystrophy (DMD).

CAP-1002 is a therapy based on cardiosphere-derived cells (CDCs), which is expected to become a treatment option for cardiomyophathy associated to Duchenne. The decision of the FDA was based on preclinical studies conducted by the company that demonstrated the potential of the therapy. The cells are able to promote cardiomyogenesis and angiogenesis, as well as inhibit oxidative stress, inflammation and fibrosis, as announced by Capricor.

“We are pleased to have received orphan drug designation for CAP-1002 for the treatment of DMD,” stated the CEO of Capricor, Linda Marbán, PhD. “Duchenne cardiomyopathy is a devastating complication of the disease that impacts virtually all those affected by the disease and is the leading cause of death in patients. We believe CDCs are uniquely suited to treat the cardiac component of DMD and could potentially be used in conjunction with any of the therapeutics currently in use or under investigation for the skeletal muscle pathology.”

The orphan drug designation is granted by the FDA’s Office of Orphan Drug Products for experimental medical therapies expected to treat rare conditions that affect no more than 200,000 people in the country. Due to the orphan drug status, Capricor is now eligible for special incentives, such as tax credits on the clinical development costs, prescription drug user fee waivers and the possibility to have seven years of market exclusivity in the U.S. after the drug is approved by the FDA.

“Capricor is addressing the devastating cardiac complications for which there is no specific treatment. We anticipate filing an IND with the FDA in the near future and hope to be treating patients with Duchenne later this year subject to regulatory approval. We look forward to reporting additional milestones as they are achieved,” added Marbán.

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The results of the pre-clinical studies were presented at the American Heart Association’s annual meeting last November, revealing an improvement in cardiac function and exercise capacity observed in mice treated with the CDC. The researchers observed attenuated inflammation, improved mitochondrial function and reduced collagen and fibrosis in the heart, suggesting the potential of CAP-1002 to treat Duchenne patients.

The Beverly Hills, California-based company had announced last October that it would start exploring one of their leading pipeline products indicated for heart disease for the treatment of complications from the extremely rare muscle-wasting disease.