Also known as: Capricor Therapeutics
Capricor Therapeutics is a biotechnology company focused on cell and exosome-based therapeutics for rare diseases. Its lead candidate is Deramiocel for Duchenne muscular dystrophy, and it is also developing exosome platforms for broader therapeutic delivery.
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
Diamyd Medical anticipates March 2026 interim results from its Phase 3 diabetes trial that could support a BLA pathway. Capricor Therapeutics awaits an August 2026 FDA decision on its Duchenne muscular dystrophy treatment BLA. Bicara Therapeutics prepares for Phase 3 study initiation of its oncology candidate toward future BLA submission.
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.
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
