FDA Resumes Review of Capricor's Deramiocel for Duchenne Muscular Dystrophy
The FDA has lifted a Complete Response Letter and resumed review of Capricor Therapeutics' Biologics License Application for deramiocel, setting a PDUFA date of August 22nd. The company ended 2025 with $318M in cash.
The Food and Drug Administration has lifted the previously issued Complete Response Letter and resumed the review of the Biologics License Application for deramiocel and set a PDUFA date of August 22nd. The cell therapy candidate targets Duchenne muscular dystrophy.
Capricor Therapeutics ended 2025 with cash of $318M to fund operations through 2027. The biopharmaceutical firm is finalizing the submission of its comprehensive HOPE-3 Phase 3 study report to the Food and Drug Administration, marking a critical juncture in the regulatory review. These documents are designed to provide the evidence of efficacy and safety required for the ongoing Biologics License Application.
The label could include the broad Duchenne muscular dystrophy population, not just DMD cardiomyopathy. This submission builds upon positive study results from late 2025 and aims to satisfy the agency's specific requirements for clinical data.
The FDA's move to tighten its expedited approval pathway directly affects Capricor Therapeutics. This places more attention on how its Duchenne muscular dystrophy program and other rare disease candidates progress through upcoming clinical and regulatory checkpoints.
Capricor's share price has moved to US$27.90 after a recent 1 day share price return of 8.06%, while its 1 year total shareholder return of 117.80% suggests momentum has been strong over a longer horizon. The company is trading at US$27.90 versus a stated price target of US$50.80.
The continued rise in the global prevalence of rare and genetic diseases is directly fueling unmet medical needs in areas like Duchenne Muscular Dystrophy, placing Capricor's Deramiocel in a strong position for regulatory support and future market demand. With growing adoption of personalized and precision medicine, Capricor's advanced exosome platform is now in the clinic with StealthX, broadening potential future applications and strategic partnerships.