Capricor Therapeutics Announces Establishment of New PDUFA Date for Deramiocel BLA
MWN-AI** Summary
Capricor Therapeutics (NASDAQ: CAPR) has announced that the U.S. Food and Drug Administration (FDA) has resumed its review of the company’s Biologics License Application (BLA) for Deramiocel, an investigational therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. This decision follows a Complete Response Letter (CRL) issued in July 2025, where no potential review issues have been indicated for the application's current evaluation. The new Prescription Drug User Fee Act (PDUFA) target action date has been set for August 22, 2026.
The BLA for Deramiocel, supported by positive data from the pivotal HOPE-3 Phase 3 trial, aims to be the first therapy to address both the skeletal and cardiac complications of DMD. Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated potent immunomodulatory and anti-fibrotic effects in clinical studies, helping to preserve muscle function in patients with DMD.
Capricor’s CEO Linda Marbán expressed confidence in the FDA's decision and the potential for Deramiocel to become a first-in-class treatment option. The company also anticipates being eligible for a Priority Review Voucher if Deramiocel is approved, further underscoring their commitment to expediting access to this crucial therapy for patients.
DMD, primarily affecting boys, leads to severe muscle degeneration and often results in heart complications due to the absence of the dystrophin protein. With approximately 15,000 individuals affected in the U.S., there are currently limited treatment options and no cure, positioning Deramiocel as a potentially transformative solution in the DMD therapeutic landscape. Capricor remains focused on advancing this promising therapy through the ongoing regulatory process.
MWN-AI** Analysis
Capricor Therapeutics (NASDAQ: CAPR) recently announced the establishment of a new Prescription Drug User Fee Act (PDUFA) date of August 22, 2026, for its Biologics License Application (BLA) for Deramiocel, a potential game-changer in the treatment of Duchenne muscular dystrophy (DMD). This update follows the FDA's lifting of a Complete Response Letter (CRL), which indicates progress in Capricor's negotiations with regulators following the positive results from the pivotal HOPE-3 Phase 3 trial.
The significance of Deramiocel lies in its dual-targeted approach, addressing both skeletal and cardiac manifestations of DMD—an area where treatment options are alarmingly limited. This therapeutic innovation offers a crucial opportunity for Capricor to establish itself as a leader in the rare disease market. Furthermore, the anticipated eligibility for a Priority Review Voucher (PRV) upon potential approval enhances the attractiveness of investing in CAPR at this juncture.
Despite the positive trajectory, investors should remain cautious given the inherent volatility in biotech stocks, especially those reliant on regulatory outcomes. The time frame until the PDUFA date allows room for fluctuation based on market sentiment, clinical data releases, and broader sector trends.
Capricor's extensive preclinical and clinical data, as well as collaborations, add credibility to its potential but investors must also consider the risks associated with FDA approvals—a process fraught with uncertainty. Hence, a balanced approach is recommended. For longer-term investors, this could serve as an attractive entry point, especially given the transformative potential of Deramiocel. However, those with short-term horizons should heed the market's intrinsic volatility and prepare for potential price swings leading up to the action date. Maintaining a diversified portfolio could mitigate risks while capitalizing on Capricor's promising pipeline.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
- 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
FAQ**
How does Capricor Therapeutics Inc. (CAPR) plan to address potential regulatory challenges as the FDA reviews Deramiocel's BLA for Duchenne muscular dystrophy?
What specific mechanisms of action do the cardiosphere-derived cells in Deramiocel employ to address both skeletal and cardiac manifestations of Duchenne muscular dystrophy, as highlighted by Capricor Therapeutics Inc. (CAPR)?
Can Capricor Therapeutics Inc. (CAPR) provide insights on how the positive HOPE-3 Phase 3 trial results influence the anticipated timeline for Deramiocel’s market approval and availability to patients?
What strategies does Capricor Therapeutics Inc. (CAPR) have in place to maximize the potential benefits of a Priority Review Voucher upon the approval of Deramiocel for Duchenne muscular dystrophy treatment?
**MWN-AI FAQ is based on asking OpenAI questions about Capricor Therapeutics Inc. (NASDAQ: CAPR).
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