Capricor Therapeutics Announces Key Regulatory Updates for its Duchenne Muscular Dystrophy Program
- FDA Pre-License Inspection completed successfully with only minor observations
- BLA under priority review with PDUFA date set for August 31, 2025
- Mid-cycle review completed with no significant issues or major deficiencies
- Multiple regulatory designations secured (Orphan Drug, RMAT, ATMP)
- Potential eligibility for Priority Review Voucher upon approval
- Exclusive commercialization agreement with Nippon Shinyaku for US and Japan markets
- Form 483 observations received during facility inspection, requiring company response
- Final facility approval still pending FDA review
- Advisory Committee meeting date still pending final FDA confirmation
Insights
Capricor's Deramiocel advances through key FDA milestones toward potential August approval for Duchenne muscular dystrophy treatment.
Capricor has reached several critical regulatory milestones for Deramiocel, its cell therapy for Duchenne muscular dystrophy (DMD). The FDA's Pre-License Inspection (PLI) was completed with only minor observations related to quality systems and documentation practices, none requiring material changes to manufacturing processes. This is a significant achievement in the biologics manufacturing space, where regulatory standards are exceptionally rigorous.
The regulatory pathway is progressing according to schedule with three key developments: (1) completion of the mid-cycle review without major deficiencies identified, (2) scheduling of an FDA Advisory Committee meeting for July 30, 2025, and (3) confirmation that the Biologics License Application (BLA) remains under priority review with a PDUFA date of August 31, 2025.
The granting of priority review indicates the FDA recognizes Deramiocel as potentially addressing an unmet medical need in a serious condition. If approved, Deramiocel would represent a novel therapeutic approach for DMD, utilizing allogeneic cardiosphere-derived cells (CDCs) that exert immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function.
The regulatory positioning is further strengthened by multiple designations: Orphan Drug status from both FDA and EMA, Regenerative Medicine Advanced Therapy (RMAT) designation in the US, and Advanced Therapy Medicinal Product (ATMP) designation in Europe. These designations provide enhanced regulatory support and potential market exclusivity. Additionally, approval would trigger eligibility for a valuable Priority Review Voucher based on the previously granted rare pediatric disease designation.
With the late-cycle meeting planned for mid-July, all major regulatory hurdles appear to be progressing favorably toward the August 31st decision date. The Advisory Committee meeting will be particularly crucial as it will publicly evaluate the risk-benefit profile of Deramiocel before making recommendations to the FDA regarding approval.
- U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support licensure
- Advisory Committee meeting scheduled for July 30, 2025
- Mid-cycle meeting recently completed with no significant issues or major deficiencies; late-cycle meeting planned for mid-July
- Biologics License Application remains under priority review with PDUFA target action date of August 31, 2025
SAN DIEGO, June 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its San Diego manufacturing facility for Deramiocel, the Company’s lead cell therapy candidate with a Biologics License Application (BLA) under FDA review for potential approval in the treatment of Duchenne Muscular Dystrophy (DMD). The inspection concluded with a Form 483 containing several observations. The Company has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. The observations were primarily related to routine quality systems and documentation practices. The Company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch.
“This inspection outcome is a major regulatory milestone, particularly in a field where standards are exceptionally high,” said Linda Marbán, Ph.D., Capricor’s Chief Executive Officer. “It reflects the strength of our manufacturing capabilities and positions us well as we advance toward potential approval. With the FDA Advisory Committee meeting now scheduled, we look forward to the opportunity to present the totality of evidence supporting the approval of Deramiocel for the treatment of Duchenne muscular dystrophy. With all key review activities progressing on track, we remain focused on delivering this much-needed therapy to the Duchenne community.”
The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major deficiencies were noted. A late-cycle meeting is planned for mid-July 2025. The BLA for Deramiocel remains under priority review with a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025.
About Duchenne Muscular Dystrophy
DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure.
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 preservation of cardiac and skeletal muscle function in dystrophiopathies 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 a 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 for the treatment of DMD has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for Deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
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. Extensive preclinical and clinical studies have shown deramiocel to exert potent immunomodulatory and anti-fibrotic actions in preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. Deramiocel is currently in late-stage development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand 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 Twitter.
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 discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; 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; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; 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 March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 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 is an Investigational New Drug (IND) and is not yet approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.
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
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