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Economics, Business & Work 2026-08-25 00:00 UTC edition Story 24 of 24 Gate: passed

FDA Extends Review Timeline for Capricor Deramiocel Application

The FDA has delayed its decision on Capricor Therapeutics' Deramiocel until November 2026 to review new clinical data regarding upper limb function in Duchenne muscular dystrophy patients.

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FDA Extends Review Timeline for Capricor’s Deramiocel BLA

The FDA pushed back its decision deadline for Capricor Therapeutics' application for Deramiocel, moving the target action date from August 22, 2026, to November 22, 2026. This three-month delay gives the agency time to review new clinical data, shifting the timeline for a potential commercial launch of a therapy aimed at a rare genetic disease.[1][2]

The delay happened because Capricor submitted a major amendment to its application. This submission includes 24-month open-label extension data from its Phase 3 HOPE-3 study. The company wants the FDA to approve the therapy specifically for improving upper limb function, which was the main focus of the trial.[3][4]

Duchenne muscular dystrophy affects about 15,000 people in the United States. Deramiocel, also known as CAP-1002, uses allogeneic cardiosphere-derived cells to help reduce inflammation and tissue scarring. The HOPE-3 trial met its primary goal, showing a clear benefit in helping patients maintain upper limb function. The FDA's Center for Biologics Evaluation and Research accepted the new data for review because of the high unmet medical need.[5][6][7][8]

Key takeaways

  • The amendment includes 24-month open-label extension data from the HOPE-3 study, providing one of the most extensive clinical datasets for upper limb function in DMD. 1 source
  • Capricor is requesting a refined indication focused on upper limb function, citing the importance of preserving independence for patients with Duchenne muscular dystrophy. 1 source
  • CEO Linda Marbán noted that the additional data strengthens the evidence supporting the therapy's clinical benefit. 1 source
  • Monitor the November 22, 2026, PDUFA date for the final regulatory decision on the Deramiocel BLA. 1 source

What’s unresolved

  • Final FDA decision on the refined indication for Deramiocel.
  • Impact of the delay on the existing distribution agreement with Nippon Shinyaku.

Citations

  1. [1] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA The FDA extended the PDUFA target action date for the Deramiocel BLA from August 22, 2026, to November 22, 2026. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  2. [2] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA The FDA classified the submission as a major amendment, necessitating a three-month extension. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  3. [3] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA Capricor submitted a major amendment to the BLA including 24-month open-label extension data from the Phase 3 HOPE-3 study. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  4. [4] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA Capricor is requesting that the FDA review data in support of a refined proposed indication focused on upper limb function. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  5. [5] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA Duchenne muscular dystrophy affects approximately 15,000 individuals in the United States. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  6. [6] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that exert immunomodulatory and anti-fibrotic actions. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  7. [7] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA HOPE-3 met its primary endpoint, demonstrating a statistically significant benefit in upper limb function. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP
  8. [8] Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA The FDA's CBER accepted the amendment for review, citing the significant unmet medical need in DMD. Manifest ID 1787604044543130285 - Capricor Therapeutics Blog Post - interrogate via MCP