STAT is live-blogging an FDA meeting on Capricor Therapeutics’ treatment for Duchenne muscular dystrophy, which has been the subject of a contentious regulatory review.
Welcome to STAT’s live blog coverage of the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee meeting, which on Wednesday is reviewing deramiocel, a cell therapy for Duchenne muscular dystrophy developed by Capricor Therapeutics.
The stakes are rather high. Capricor has positioned deramiocel as one of the only treatment options for teenagers and young men with Duchenne who have already lost their ability to walk. In a Phase 3 study, deramiocel preserved upper-arm function and staved off the heart failure most patients eventually experience, Capricor said last December, achieving the primary and secondary goals of the trial with statistical significance.
The FDA, in a harshly critical review posted publicly on Monday, disagreed with the company’s conclusions. In its own analysis, deramiocel failed to separate from placebo on the upper-limb and cardiac function endpoints, agency reviewers concluded. Furthermore, Capricor made numerous changes to the deramiocel study’s statistical analysis plan after it was completed, further muddying the outcome, the FDA said.