FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee didn’t hedge on July 29: 9-3 against, ruling Capricor Therapeutics’ deramiocel ineffective for Duchenne muscular dystrophy cardiomyopathy, per the committee’s Federal Register notice convening the BLA review. CEO Linda Marbán won’t rule out suing the agency over it.
I read the underlying fight, and it comes down to which statistical plan counts as the benchmark. FDA anchored its p-value analysis to SAP 1.1, which Marbán calls an unsigned, incomplete draft that predated the trial’s second cohort, instead of SAP 3.0, the version Capricor finalized before unblinding. Old yardstick beat new yardstick, and the primary endpoint lost significance because of it. “They just kind of threw it all together in a crockpot and hoped for dinner,” Marbán said. FDA disputes the singular focus, saying its review covered “several versions” of the plan.
Shares cratered nearly 80% over five days before a Thursday rebound closed them at $4.19, still down from $6.69 pre-meeting. The timing stings: the Lancet’s HOPE-3 publication hit the wires minutes into the meeting, showing deramiocel slowed upper limb decline 54% versus placebo.
This isn’t Duchenne’s first benchmark fight this year. FDA’s 2025 Elevidys shipment pause after patient deaths in Sarepta’s gene therapy already had the space on edge, and Marbán flagged that history before Wednesday’s meeting even happened. Extra caution explains some of it. A statistical plan finalized one day before unblinding explains more.
FDA’s PDUFA date for deramiocel is August 22. Worth reading the SAP 1.1-versus-3.0 argument before that date lands, because it’s the fight every DMD sponsor with a long trial will eventually have.
— Rebecca Lauren