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.
