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Jump to Key Points Aug 24 (Reuters) - Capricor Therapeutics said on Monday the U.S. Food and Drug Administration had extended the review period for its experimental cell therapy for Duchenne muscular dystrophy by three months.
The delay follows Capricor's submission of new 24-month data from its late-stage study, along with extra analyses, to support a refined proposed indication focused on upper limb function, the trial's main goal.
The therapy, deramiocel, last year failed to get FDA's approval to treat cardiomyopathy, or a heart muscle disease, in patients with Duchenne muscular dystrophy. The regulator said the treatment did not meet efficacy requirements and asked for more data.
Earlier this year, Capricor resubmitted its application with new data, and was expecting a decision by August 22.
- FDA extended deramiocel review 3 months; new decision date Nov 22.
- Amendment included 24-month data + extra analyses for refined upper-limb indication.
- Advisory panel voted against efficacy data; FDA staff noted endpoint-metric changes.
- Prior FDA rejection cited inadequate efficacy for Duchenne cardiomyopathy.
- Duchenne muscular dystrophy affects ~15,000 U.S. patients, mainly boys.
But last month, an FDA advisory panel voted against the therapy's effectiveness data, while the agency staff flagged concerns about changes to how the company measured trial results.
Following this, Capricor Chief Executive Linda Marban said the company will submit an amendment with new data focused on improving upper limb muscle function.
The health regulator has accepted the amendment, citing the high unmet medical need for Duchenne patients, and classified the submission as a "major amendment" and set the new deadline to November 22, Capricor said.
Shares of the company rose 15% to $7.25.
Marbán said the additional follow-up data gives the company "one of the most extensive clinical datasets evaluating upper limb function in Duchenne," and that the trial had met its main goal with a statistically significant benefit.
Deramiocel is being developed to treat Duchenne, a rare genetic disease that affects about 15,000 people in the United States, primarily boys.
The key question remains whether the new data and analyses are sufficient to "address the FDA's concerns and support approval for the refined indication focused on upper limb function," said H.C. Wainwright analysts.
(Reporting by Kamal Choudhury in Bengaluru; Editing by Shinjini Ganguli)
