FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) has received FDA acceptance for its Biologics License Application (BLA) for deramiocel, an investigational cell therapy targeting Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA has granted Priority Review with a PDUFA target action date of August 31, 2025.

NS Pharma, a subsidiary of Nippon Shinyaku, will be exclusively responsible for the U.S. commercialization and distribution of deramiocel, following their exclusive distribution agreement established in January 2022. The FDA has not identified any potential review issues at this time.

Positive

• FDA acceptance of BLA with Priority Review status
• No potential review issues identified by FDA
• Exclusive U.S. distribution agreement with established pharma company NS Pharma
• Addressing unmet medical need in DMD market

Negative

• None.

PARAMUS, N.J., March 10, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that acceptance has been received by Capricor Therapeutics, Inc. (Headquarters: California, USA, CEO: Linda Marbán, NASDAQ: CAPR) from the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) filing for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy ("DMD") cardiomyopathy. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of August 31, 2025, and at this time, the FDA has not identified any potential review issues.

"Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD."

Nippon Shinyaku and Capricor entered into an exclusive distribution agreement for deramiocel for the U.S. in January 2022. NS Pharma will be exclusively responsible for commercialization and distribution of deramiocel in the U.S.

"Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD," said NS Pharma President, Yukiteru Sugiyama, Ph.D. "We are excited for the possibility to bring additional treatment options and renewed hope to families of the rare disease community."

For more details, please see the press release from Capricor.
https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority

About Deramiocel
Deramiocel consists of allogeneic cardiosphere-derived cells ("CDCs"), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.

About Duchenne Muscular Dystrophy (Duchenne)
Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive weakness and loss of skeletal, cardiac, and respiratory muscles. Early signs of Duchenne may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with Duchenne may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. For more information about Duchenne, please visit wespeakduchenne.com.

About Capricor Therapeutics, Inc.
Capricor (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. 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. For more information, https://www.capricor.com.

About NS Pharma, Inc. NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. NS Pharma is a registered trademark of the Nippon Shinyaku Co., Ltd. For more information, please visit nspharma.com.

US Media Contact:
media@nspharma.com

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SOURCE NS Pharma, Inc.

FAQ

When is the FDA PDUFA date for Capricor's (CAPR) deramiocel DMD treatment?

The FDA PDUFA target action date for Capricor's deramiocel is August 31, 2025.

What type of FDA review did Capricor (CAPR) receive for its DMD treatment?

Capricor received Priority Review from the FDA for deramiocel's Biologics License Application.

Who will distribute Capricor's (CAPR) deramiocel in the United States?

NS Pharma will exclusively handle the U.S. commercialization and distribution of deramiocel.

What condition does Capricor's (CAPR) deramiocel treat?

Deramiocel is designed to treat cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).