FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment
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.
Capricor Therapeutics (NASDAQ: CAPR) ha ricevuto l'accettazione da parte della FDA per la sua domanda di licenza biologica (BLA) per deramiocel, una terapia cellulare sperimentale mirata alla cardiomiopatia da distrofia muscolare di Duchenne (DMD). La FDA ha concesso una revisione prioritaria con una data obiettivo di azione PDUFA fissata per il 31 agosto 2025.
NS Pharma, una sussidiaria di Nippon Shinyaku, sarà esclusivamente responsabile della commercializzazione e distribuzione di deramiocel negli Stati Uniti, in seguito all'accordo di distribuzione esclusivo stabilito a gennaio 2022. Al momento, la FDA non ha identificato alcun problema potenziale di revisione.
Capricor Therapeutics (NASDAQ: CAPR) ha recibido la aceptación de la FDA para su Solicitud de Licencia Biológica (BLA) para deramiocel, una terapia celular en investigación dirigida a la cardiomiopatía por distrofia muscular de Duchenne (DMD). La FDA ha otorgado Revisión Prioritaria con una fecha de acción objetivo PDUFA del 31 de agosto de 2025.
NS Pharma, una subsidiaria de Nippon Shinyaku, será responsable exclusivamente de la comercialización y distribución de deramiocel en EE. UU., tras su acuerdo de distribución exclusiva establecido en enero de 2022. En este momento, la FDA no ha identificado ningún problema potencial de revisión.
Capricor Therapeutics (NASDAQ: CAPR)는 두셴 근육 위축증(DMD) 심근병증을 목표로 하는 실험적인 세포 치료제인 deramiocel에 대한 생물학적 라이센스 신청(BLA)이 FDA의 승인을 받았다고 발표했습니다. FDA는 우선 검토를 승인했으며, PDUFA 목표 조치 날짜는 2025년 8월 31일로 설정되었습니다.
NS Pharma는 2022년 1월에 체결된 독점 배급 계약에 따라 deramiocel의 미국 내 상업화 및 배급을 독점적으로 책임지게 됩니다. 현재로서는 FDA가 검토 문제를 식별하지 않았습니다.
Capricor Therapeutics (NASDAQ: CAPR) a reçu l'acceptation de la FDA pour sa demande de licence biologique (BLA) pour deramiocel, une thérapie cellulaire expérimentale ciblant la cardiomyopathie liée à la dystrophie musculaire de Duchenne (DMD). La FDA a accordé un examen prioritaire avec une date d'action cible PDUFA fixée au 31 août 2025.
NS Pharma, une filiale de Nippon Shinyaku, sera exclusivement responsable de la commercialisation et de la distribution de deramiocel aux États-Unis, suite à leur accord de distribution exclusif établi en janvier 2022. À ce jour, la FDA n'a identifié aucun problème potentiel de révision.
Capricor Therapeutics (NASDAQ: CAPR) hat die Zustimmung der FDA für seinen Antrag auf biologische Lizenz (BLA) für deramiocel erhalten, eine experimentelle Zelltherapie, die auf die kardiomyopathische Form der Duchenne-Muskeldystrophie (DMD) abzielt. Die FDA hat eine priorisierte Überprüfung gewährt, mit einem PDUFA-Zieltermin für die Entscheidung am 31. August 2025.
NS Pharma, eine Tochtergesellschaft von Nippon Shinyaku, wird nach dem im Januar 2022 geschlossenen exklusiven Vertriebsvertrag ausschließlich für die Kommerzialisierung und den Vertrieb von deramiocel in den USA verantwortlich sein. Derzeit hat die FDA keine potenziellen Prüfungsprobleme identifiziert.
- 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
- None.
Nippon Shinyaku and Capricor entered into an exclusive distribution agreement for deramiocel for the
"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.
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