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Capricor Duchenne cell therapy voted down by FDA panel
['Ben Fidler', 'Lead Editor']
BioPharma Dive - Latest News
Dive Brief:Capricor Therapeutics hasn’t definitively proven that its experimental cell therapy benefits people with heart-related complications of Duchenne muscular dystrophy, a Food and Drug Administration panel concluded Wednesday.
Capricor reported last year that its treatment, deramiocel, hit the main goals of a Phase 3 study in Duchenne patients with cardiomyopathy.
The FDA rejected deramiocel last July but agreed to review a revised pitch from Capricor afterwards.
However, briefing documents submitted by FDA scientists ahead of the meeting revealed another disconnect between the Capricor and the agency.
FDA reviewers argued the statistical changes Capricor employed “lack a scientific basis” and made the findings difficult to interpret.