Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and outgoing Vinay Prasad, director of the Center for Biologics Evaluation and Research.
Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy is back on track after the FDA announced that it will once again review the biotech’s application package.
The agency has lifted its previous complete response letter on the cell therapy, dubbed deramiocel, Capricor revealed in a news release on Tuesday. The biotech is seeking full approval for deramiocel, for which the FDA’s verdict is now expected on Aug. 22. The agency has yet to identify any potential review issues that deramiocel could encounter, according to Capricor.
Capricor’s shares were up 9% to $33.40 as of market close Tuesday after the announcement.
Composed of cardiosphere-derived cells—a rare subset of cells from the heart that can modulate the immune response and reduce fibrosis—deramiocel is an allogenic cell therapy that helps preserve the function of cardiac and skeletal muscles. Capricor tested the therapy in the Phase 2 HOPE-2 study, and in May 2020 reported that it elicited meaningful improvements in upper limb function in patients with Duchenne muscular dystrophy (DMD).
HOPE-2 became the basis of Capricor’s initial approval package for deramiocel, which the FDA turned down in July 2025 claiming that the submission lacked the “statutory requirement for substantial evidence of effectiveness.”
At the time, observers suggested that deramiocel might have been caught in the crossfire between two top figures in the FDA: Nicole Verdun, former top regulator of cell and gene therapies at the Center for Biologics Evaluation and Research (CBER), who wanted to hold an advisory committee meeting for the asset, and Vinay Prasad, current director of the Center for Drug Evaluation and Research (CDER), who unilaterally canceled the meeting. Verdun was ultimately pushed out of the agency in June 2025.
Prasad is once again set to leave the FDA by the end of April, a move that analysts say could be a tailwind for the rare disease space—including Capricor.
In December 2025, Capricor resubmitted its drug package for deramiocel, beefing it up with data from the Phase 3 HOPE-3 trial. The study found that patients given the cell therapy saw significant improvements in upper-limb function and cardiac function compared with those on placebo. Deramiocel also slowed skeletal muscle disease progression.
If approved, Capricor could receive a priority review voucher under the FDA’s rare pediatric disease program, the biotech said on Tuesday.
