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Capricor Provides Regulatory Update On Deramiocel BLA For Duchenne Muscular Dystrophy

By RTTNews Staff Writer   ✉   | Published:   | Follow Us On Google News

Capricor Therapeutics (CAPR) on Thursday announced a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).

The update follows a recent Type A meeting with the U.S. Food
and Drug Administration (FDA) after the receipt of a Complete Response Letter (CRL) in July 2025.

Key outcomes from the meeting include confirmation that the HOPE-3 trial will fulfill the requirement for the additional study outlined in the CRL, and that the HOPE-3 data can be submitted within the current BLA.

The company also noted that the FDA and Capricor have aligned on the endpoints for the HOPE-3 pivotal trial. Capricor plans to submit the HOPE-3 data, expected in the fourth quarter of this year, to support the BLA resubmission.

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