Capricor Therapeutics Announces Orphan Drug Designation for Becker Muscular Dystrophy and Regulatory Progress for Duchenne Muscular Dystrophy Program
- U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor's focus in neuromuscular diseases
- Capricor remains on track for the August 31, 2025, PDUFA date for Deramiocel in Duchenne Muscular Dystrophy following successful FDA Pre-License Inspection
SAN DIEGO, June 17, 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 that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel, the company's lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor's strategic position as it advances a fully integrated platform targeting the cardiac and skeletal complications of muscular dystrophy and expands the commercial potential of its lead asset.
Becker Muscular Dystrophy, like Duchenne Muscular Dystrophy (DMD), is a progressive X-linked neuromuscular disorder that results in significant skeletal and cardiac muscle deterioration over time. Deramiocel is being developed to address both aspects of disease pathology, including cardiomyopathy, a primary contributor to morbidity and mortality in patients with both BMD and DMD.
"The granting of Orphan Drug Designation for Becker Muscular Dystrophy is a significant milestone that expands the potential reach of Deramiocel and reinforces our vision to deliver impactful therapies across a broader spectrum of neuromuscular diseases," said Linda Marbán, Ph.D., CEO of Capricor Therapeutics. "We believe Deramiocel holds promise for patients with Becker, particularly given the overlap in disease pathology with Duchenne. Deramiocel has demonstrated potential to treat the serious cardiac and skeletal muscle complications of Duchenne Muscular Dystrophy, and based on the overlap in disease pathology, may also offer benefit to patients with Becker Muscular Dystrophy."
Dr. Marbán continued, "In addition, Capricor successfully completed its Pre-License Inspection, an important regulatory milestone for approval of its Biologics License Application (BLA) for DMD and we believe all review activities remain on track as we approach our PDUFA date. With key milestones aligning, we continue to prepare for the potential commercial launch of Deramiocel and the opportunity to deliver meaningful benefits to patients with Duchenne."
Capricor's Biologics License Application (BLA) for Deramiocel in DMD remains under priority review, with a PDUFA target action date of August 31, 2025.
About Duchenne and Becker 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.
Becker Muscular Dystrophy (BMD) is a related dystrophinopathy caused by mutations in the same gene as DMD. It typically presents later in life with a slower disease progression and affects an estimated 5,000 individuals in the United States. Despite its milder course, many BMD patients develop serious cardiac complications, including cardiomyopathy, which can significantly affect both quality of life and longevity.
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 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 has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (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. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the FDA.
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 the preservation of cardiac and skeletal muscle function in muscular dystrophies 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. Neither BMD nor any of Capricor's exosome-based candidates have been approved for clinical use.
For more information, please contact:
Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
[email protected]
212.896.1204
Capricor Company Contact:
AJ Bergmann, Chief Financial Officer
[email protected]
858.727.1755
