The FDA refuses Capricor’s cell therapy for cardio complications of rare muscular diseases

Capricor Therapeutics cell therapy for Duchenne muscular dystrophy failed FDA, the regulator rejecting the application of biotechnology in search of regulatory approval of what the company hoped to become the first treatment developed specifically for the cardiovascular complications of this rare disease.

The FDA complete letter of response indicated that the request for therapy, named Deramiocel, lacks substantial evidence of efficiency and needs additional clinical data, Capricor, based in San Diego, said on Friday. The submission was based on the data of an open phase 2 test. A phase 3 study controlled by placebo is underway; The preliminary results are expected later in the current quarter. Capricor CEO Linda Marbán said that the company planned to submit a therapy request with Phase 3 Study data to provide the additional evidence that the FDA needs.

“We believe that these data, if they are positive, as well as our existing long -term clinical results showing cardiac stabilization, preserving the function of skeletal muscles and a coherent security profile, could support efforts to resolve the questions raised by the FDA for the treatment of cardiomyopathy associated with [Duchenne muscular dystrophy]”Said Marbán in a prepared statement.

Duchenne is a hereditary disorder that leads to a dystrophin deficiency, a key muscle protein. Although the disease is initially manifested as gradually aggravating the weakness of the skeletal muscles, it also affects heart muscle, causing cardiomyopathy and heart failure. Capricor’s deraamil is standard therapy from donor heart fabric. Cardiospheres, a rare type of heart cell, are isolated from donor samples. Administered to a duchenne patient, these cells secrete tiny vesicles called exosomes that target macrophages, a type of immune cell. Capricor says that these cells have therapeutic effects, including the fight against inflammation and fibrosis in the heart tissue.

In March, the FDA accepted the Capricor Biology License Application for Deramicel under priority review, setting a target date of August 31 for a regulatory decision. But agency leaders have changed since then. Peter Marks, director of the Center for Biologics Evaluation and Research of the FDA, suddenly left the FDA in April after being faced with the Secretary of Health and Social Services, Robert F. Kennedy Jr. Marks, was succeeded in Vinay Prasad, a hematologist-oncologist who was critical due to certain applications of the FDA.

In May, Capricor said that the FDA would convene a meeting of the Advisory Committee to discuss cell therapy. But at the end of June, the FDA informed Capricor that this advisory meeting was no longer necessary. This announcement occurred a few days after Nicole Verdun, director of the office of therapeutic products within the Center for Biologics Evaluation and Research (CBER) of the agency, was put on administrative leave. Prasad was skeptical about Capricor’s cell therapy and unilaterally canceled the consultative meeting, said the health information site reported at the time, citing an anonymous source familiar with the issue.

In Capricor’s Friday announcement, Marbán qualified FDA’s decision on Deramiocel as a surprise. Before the complete letter of response, the Deramiocel exam went without major problems, she said. The letter also referred to questions in the chemistry, manufacturing and application control section. The company considers that it has addressed these questions, but these answers were not officially examined by the agency due to the complete letter of response. Capricor plans to request a meeting with the FDA to discuss the next steps.

Investors have gathered in the rejection of the FDA of Deramiocel. The Capricor equity price opened its doors on Friday at $ 6.99, down 38.6% compared to the closing price on Thursday.

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