Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA
Rhea-AI Summary
Capricor Therapeutics (NASDAQ: CAPR) announced the FDA has lifted its Complete Response Letter and resumed review of the Deramiocel BLA as a Class 2 resubmission, setting a PDUFA target action date of August 22, 2026. The company expects potential eligibility for a Priority Review Voucher upon approval.
Deramiocel is supported by positive HOPE-3 Phase 3 results and holds multiple designations including Orphan Drug, RMAT, ATMP, and Rare Pediatric Disease.
Positive
- FDA resumed BLA review and set a Aug 22, 2026 PDUFA target action date
- HOPE-3 Phase 3 reported achievement of the primary endpoint and all Type I error-controlled secondary endpoints
- Deramiocel holds multiple regulatory designations (Orphan Drug, RMAT, ATMP, Rare Pediatric Disease)
- Potential eligibility for a Priority Review Voucher upon approval
Negative
- Company previously received a Complete Response Letter in July 2025, indicating earlier deficiencies
- Approval remains uncertain until FDA’s final decision on Aug 22, 2026
News Market Reaction – CAPR
In the Mar 10 session, CAPR gained 9.04%, reflecting a notable positive market reaction. Argus tracked a peak move of +14.2% during that session. Our momentum scanner triggered 27 alerts that day, indicating elevated trading interest and price volatility. Trading volume was above average at 1.9x the daily average, suggesting increased trading activity.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Mar 09 | Earnings call notice | Neutral | +9.8% | Scheduled Q4 and full-year 2025 results call highlighting Deramiocel and StealthX. |
| Feb 24 | Clinical data update | Positive | +0.1% | HOPE-3 Phase 3 results selected for late-breaking talk and CSR submitted to FDA. |
| Jan 20 | Regulatory update | Positive | -0.6% | FDA requested HOPE-3 CSR but no new studies, supporting continued BLA review. |
| Dec 16 | Clinical results webinar | Positive | +0.5% | Webinar to review positive Phase 3 HOPE-3 topline results in DMD. |
| Dec 05 | Equity offering | Negative | +5.9% | Priced $150M common stock offering at $25 per share under Form S-3 shelf. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Recent Deramiocel regulatory and clinical updates often produced modest moves, while the December $150 million equity offering saw a stronger positive reaction, indicating investors have been willing to add risk around key milestones.
Over the last several months, CAPR has focused on advancing Deramiocel toward approval. Updates on HOPE-3 data and FDA interactions on Jan 20, 2026 and Feb 24, 2026 centered on submitting the full HOPE-3 CSR and seeking a new PDUFA date. A December 2025 equity offering raised $150 million under an effective Form S-3 shelf. Today’s announcement that the FDA lifted the Complete Response Letter and set an Aug 22, 2026 PDUFA date directly follows that regulatory path.
Key Terms
pdufa regulatory
biologics license application regulatory
complete response letter regulatory
orphan drug designation regulatory
regenerative medicine advanced therapy regulatory
advanced therapy medicinal product regulatory
rare pediatric disease designation regulatory
exosomes medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
- Deramiocel has the potential to become the first therapy to address both skeletal and cardiac manifestations of Duchenne muscular dystrophy
- BLA supported by positive pivotal HOPE-3 Phase 3 results, including achievement of the primary endpoint and all Type I error-controlled secondary endpoints
- PDUFA target action date set for August 22, 2026; Company expects to be eligible for a Priority Review Voucher upon potential approval
SAN DIEGO, March 10, 2026 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application (“BLA”) seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy (“DMD”) cardiomyopathy. The submission has been classified as a Class 2 resubmission, with a Prescription Drug User Fee Act (“PDUFA”) target action date of August 22, 2026.
“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,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “We believe the positive HOPE-3 results and broader clinical evidence 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. We look forward to continuing to work closely with the FDA throughout the review process and remain focused on bringing this important therapy to patients as expeditiously as possible.”
The Company received a Complete Response Letter (“CRL”) from the FDA in July 2025. Following submission of data and supporting documentation from the HOPE-3 clinical trial, the FDA resumed review of the application and assigned a PDUFA target action date of August 22, 2026. At this time, the FDA has not identified any potential review issues in its response to the Company.
Capricor also expects to be eligible to receive a Priority Review Voucher (“PRV”) upon potential approval of Deramiocel.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain limited.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile to adopt a healing rather than pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials.
Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval.
About Capricor Therapeutics
Capricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, Deramiocel, an allogeneic cardiac-derived cell therapy that is currently in late-stage development for the treatment of Duchenne muscular dystrophy (DMD). Extensive preclinical and clinical data have demonstrated Deramiocel’s potent immunomodulatory and anti-fibrotic effects in helping to preserve cardiac and skeletal muscle function in DMD. Capricor is also leveraging the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on vaccinology and the targeted delivery of oligonucleotides, proteins, and small-molecule therapeutics, with the potential to treat and prevent a wide range of diseases. At Capricor, we are committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor on Facebook, Instagram and X.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor’s product candidates; the initiation, conduct, size, timing and results of clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including future interactions with regulatory authorities and the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; the potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; the ability to achieve product milestones and to receive milestone payments from commercial partners; and any other statements about Capricor’s management team’s future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words “believes,” “plans,” “could,” “anticipates,” “expects,” “estimates,” “should,” “target,” “will,” “would” and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor’s business is set forth in Capricor’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, as filed with the Securities and Exchange Commission on November 10, 2025. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.
Capricor has entered into an agreement for the exclusive commercialization and distribution of Deramiocel for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel and the StealthX™ vaccine are investigational candidates and have not been approved for commercial use in any indication.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
