Capricor's Duchenne drug gets three-month FDA extension after new data submission
CAPR•Analyst view and disease context
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.
New deadline and market reaction
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.
FDA extends review period for deramiocel
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.
Background on the therapy and previous FDA feedback
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.
