Deramiocel slowed Duchenne muscular dystrophy progression over five years, maintained stable cardiac function and showed a favorable safety profile after more than 800 intravenous infusions. The FDA will convene an advisory committee on July 29 to review the biologics license application ahead of the August 22, 2026 PDUFA action date.
The Hope-2 open-label extension study demonstrated that Deramiocel continued to slow decline in upper limb function over five years, with patients exhibiting stable cardiac parameters. More than 800 intravenous infusions were administered without any new safety concerns, reinforcing the therapy’s long-term tolerability.
The FDA has scheduled an advisory committee meeting for July 29 to evaluate the biologics license application for Deramiocel. The BLA remains on track for a PDUFA action date of August 22, 2026, which will determine whether the therapy can be marketed in the United States.
Shares of Capricor fell 11% following the announcement of the advisory committee meeting, reflecting investor caution over regulatory hurdles. Retail sentiment shifted to extremely bullish before the slide, with debate over whether panel input will accelerate or delay approval.
Capricor plans to present the long-term Hope-2 results alongside Phase 3 HOPE-3 data at the Parent Project Muscular Dystrophy 2026 Annual Conference. These presentations aim to bolster clinical understanding and stakeholder confidence ahead of the FDA decision.
