Capricor Updates Deramiocel BLA Status After FDA Review of HOPE-3 Data

Capricor Therapeutics has provided an update on its Biologics License Application (BLA) for Deramiocel, an innovative cell therapy for Duchenne muscular dystrophy (DMD). This announcement comes in the wake of feedback from the U.S. Food and Drug Administration (FDA) on data from the Phase 3 HOPE-3 clinical study.

FDA’s Review Process for HOPE-3 Data

In late 2025, Capricor submitted topline results from the HOPE-3 study to the FDA. Following its evaluation, the FDA requested the complete clinical study report (CSR) and additional data to address a Complete Response Letter (CRL) issued in July 2025. Crucially, no further clinical studies or new patient data have been requested by the FDA at this stage.

Next Steps for Capricor

Preparation of the CSR is currently in progress, with plans to submit the required documents by February 2026. The company anticipates that this submission will help fulfill the FDA’s requirements and facilitate the continuation of the BLA review process. This includes the assignment of a new Prescription Drug User Fee Act (PDUFA) action date.

Highlighted Results from HOPE-3 Trial

Linda Marbán, Ph.D., CEO of Capricor, expressed optimism regarding the FDA’s request for the HOPE-3 study report. The trial demonstrated significant improvements in skeletal muscle and cardiac function, which are critical for DMD patient care.

• Results have shown statistically significant advancements in muscle function.
• These findings underscore the potential of Deramiocel in addressing long-term outcomes for DMD patients.

Understanding Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a severe genetic disorder that leads to progressive muscle degeneration, particularly affecting boys and affecting approximately 15,000 individuals in the United States. The primary cause of mortality in DMD patients is heart muscle deterioration, resulting in cardiomyopathy and heart failure.

About Deramiocel

Deramiocel (CAP-1002) consists of cardiosphere-derived cells (CDCs) that have shown promise in previous studies for their anti-fibrotic and immunomodulatory properties. These cells can help preserve muscle function in conditions such as DMD by interacting with the immune system.

Regulatory Designations

Deramiocel has received multiple designations from regulatory authorities, including:

• Orphan Drug Designation from the FDA and EMA.
• Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S.
• Advanced Therapy Medicinal Product (ATMP) in Europe.
• Rare Pediatric Disease Designation from the FDA.

Conclusion

Capricor is committed to advancing its innovative therapies, particularly Deramiocel, to provide treatment options for patients with limited alternatives. The company hopes that by working closely with the FDA, it can expedite the development process for the benefit of those affected by Duchenne muscular dystrophy.

For more updates about Capricor Therapeutics and Deramiocel, visit Filmogaz.com.