Capricor Therapeutics (NASDAQ: CAPR) reported that the U.S. Food and Drug Administration lifted the Complete Response Letter issued in July 2025 and has resumed review of its Biologics License Application for Deramiocel, the company’s investigational cell therapy for Duchenne muscular dystrophy cardiomyopathy. The resubmission is classified as a Class 2 resubmission and carries a new Prescription Drug User Fee Act target action date of August 22 2026, giving the FDA a six‑month review window under the agency’s regulatory flexibility framework.
The lifting of the CRL removes a major regulatory hurdle and positions Capricor to potentially secure an $80 million milestone payment and a Priority Review Voucher upon approval. These incentives represent significant non‑dilutive capital that could support future development and commercialization activities. Deramiocel’s dual‑action profile—targeting both skeletal and cardiac manifestations of Duchenne muscular dystrophy—makes it a first‑in‑class candidate in a high‑need therapeutic area.
The FDA’s decision was driven by data from the HOPE‑3 Phase 3 trial, which met its primary endpoint and all Type I error‑controlled secondary endpoints. The trial demonstrated a 54 % slowing of progression in the PUL v2.0 skeletal muscle function score and a 91 % slowing of decline in left ventricular ejection fraction, directly addressing the evidence gaps cited in the July 2025 CRL. The robust clinical outcomes provided the FDA with the substantial evidence needed to move forward with the review.
CEO Linda Marbán said, “We are encouraged by the FDA’s acknowledgment of our response to the Complete Response Letter and its continued review of our BLA for Deramiocel.” She added that the HOPE‑3 data “reinforce Deramiocel’s potential to become a first‑in‑class therapy for Duchenne muscular dystrophy, with the opportunity to address both skeletal and cardiac manifestations of the disease.”
Analysts have maintained a strong buy consensus on Capricor, citing the regulatory progress and the compelling clinical data from the HOPE‑3 trial as key drivers of the company’s future growth prospects.
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