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FDA sets 22 November PDUFA date for Capricor's Duchenne cell therapy deramiocel after major amendment

🇺🇸 Capricor Therapeutics, Inc. (CAPR) · Tue 10 Nov 2026

The agency extended its review of the BLA after classing August's two-year HOPE-3 data and a narrower upper-limb indication as a major amendment, as Capricor reported a wider third-quarter loss.

Capricor Therapeutics said on 10 November 2026 that the US Food and Drug Administration has set a PDUFA goal date of 22 November 2026 for its biologics licence application for deramiocel, a cell therapy for Duchenne muscular dystrophy. The date follows an extension of the review.

According to the company, the extension came after the FDA classified an August submission as a major amendment. That submission contained two-year open-label data from the HOPE-3 study and a narrower proposed indication focused on the upper limb.

Capricor also disclosed that the FDA carried out a bioresearch monitoring (BIMO) inspection in July. It resulted in a Form 483 with a single observation. The summary on file gives no further detail on the observation or on how the company has responded.

In its third-quarter 2026 results, Capricor reported a net loss of $42.7 million and cash of about $198.5 million. The summary describes the loss as wider than before, but gives no comparative figures.

What it means for your team

  • A major amendment typically triggers a review-clock extension in the US; sponsors changing indications or adding datasets late in review should expect timelines to move.
  • Narrowing a proposed indication late in the process, alongside new long-term open-label data, can reshape the benefit-risk discussion with the agency.
  • A BIMO inspection with a Form 483 observation is a reminder that clinical-site and sponsor data-integrity compliance can feature in the review of cell and gene therapy applications.
  • Market-access teams tracking Duchenne therapies should note the 22 November decision date, though the outcome is not yet known.

Company's press release (SEC filing): Capricor Therapeutics, Inc. ↗

Article written by VigiEye from the official source (Capricor Therapeutics, Inc.). Claims are the company's own. AI-assisted; check the source before acting.

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