Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) announced FDA acceptance and Priority Review of its Biologics License Application (BLA) for deramiocel, a cell therapy treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA set a PDUFA target action date of August 31, 2025.
If approved, deramiocel would be the first therapy specifically targeting DMD cardiomyopathy. The treatment would be administered quarterly as a lifelong therapy. The BLA submission is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, compared with FDA-funded natural history data.
The FDA has not identified any potential review issues and has not yet decided on the need for an Advisory Committee meeting. Deramiocel has received Orphan Drug Designation from both FDA and EMA, along with RMAT designation in the U.S. and ATMP Designation in Europe.
Capricor Therapeutics (NASDAQ: CAPR) ha annunciato l'accettazione da parte della FDA e la Revisione Prioritaria della sua Domanda di Licenza Biologica (BLA) per deramiocel, un trattamento di terapia cellulare per la cardiomiopatia associata alla distrofia muscolare di Duchenne (DMD). La FDA ha fissato una data obiettivo di azione PDUFA per il 31 agosto 2025.
Se approvato, deramiocel sarebbe la prima terapia specificamente mirata alla cardiomiopatia DMD. Il trattamento verrebbe somministrato trimestralmente come terapia a vita. La presentazione della BLA è supportata da dati provenienti dagli studi di Fase 2 HOPE-2 e HOPE-2 Open Label Extension, in confronto ai dati sulla storia naturale finanziati dalla FDA.
La FDA non ha identificato potenziali problemi di revisione e non ha ancora deciso sulla necessità di una riunione del Comitato Consultivo. Deramiocel ha ricevuto la Designazione di Farmaco Orfano sia dalla FDA che dall'EMA, insieme alla designazione RMAT negli Stati Uniti e alla Designazione ATMP in Europa.
Capricor Therapeutics (NASDAQ: CAPR) anunció la aceptación por parte de la FDA y la Revisión Prioritaria de su Solicitud de Licencia Biológica (BLA) para deramiocel, un tratamiento de terapia celular para la cardiomiopatía asociada a la distrofia muscular de Duchenne (DMD). La FDA ha establecido una fecha objetivo de acción PDUFA para el 31 de agosto de 2025.
Si se aprueba, deramiocel sería la primera terapia específicamente dirigida a la cardiomiopatía DMD. El tratamiento se administraría trimestralmente como terapia de por vida. La presentación de la BLA está respaldada por datos de los ensayos de Fase 2 HOPE-2 y HOPE-2 Open Label Extension, en comparación con los datos de historia natural financiados por la FDA.
La FDA no ha identificado problemas potenciales de revisión y aún no ha decidido sobre la necesidad de una reunión del Comité Asesor. Deramiocel ha recibido la Designación de Medicamento Huérfano tanto de la FDA como de la EMA, junto con la designación RMAT en EE. UU. y la Designación ATMP en Europa.
Capricor Therapeutics (NASDAQ: CAPR)는 deramiocel에 대한 생물학적 제품 허가 신청(BLA)이 FDA의 수용 및 우선 심사를 받았다고 발표했습니다. 이는 듀센 근육디스트로피(DMD) 심근병증을 위한 세포 치료제입니다. FDA는 2025년 8월 31일을 PDUFA 목표 행동 날짜로 설정했습니다.
승인될 경우, deramiocel은 DMD 심근병증을 특별히 겨냥한 첫 번째 치료제가 될 것입니다. 이 치료는 평생 치료로서 분기별로 시행될 것입니다. BLA 제출은 FDA가 자금을 지원한 자연사 데이터와 비교하여 2상 HOPE-2 및 HOPE-2 오픈 레이블 연장 시험의 데이터로 뒷받침됩니다.
FDA는 잠재적인 검토 문제를 확인하지 않았으며, 자문 위원회 회의의 필요성에 대해서도 아직 결정하지 않았습니다. Deramiocel은 FDA와 EMA 모두에서 고아약 지정과 함께 미국에서 RMAT 지정을, 유럽에서 ATMP 지정을 받았습니다.
Capricor Therapeutics (NASDAQ: CAPR) a annoncé l'acceptation par la FDA et la Révision Prioritaire de sa Demande de Licence Biologique (BLA) pour deramiocel, un traitement par thérapie cellulaire pour la cardiomyopathie associée à la dystrophie musculaire de Duchenne (DMD). La FDA a fixé une date cible d'action PDUFA au 31 août 2025.
Si approuvé, deramiocel serait la première thérapie spécifiquement ciblant la cardiomyopathie DMD. Le traitement serait administré trimestriellement en tant que thérapie à vie. La soumission de la BLA est soutenue par des données provenant des essais de Phase 2 HOPE-2 et HOPE-2 Open Label Extension, comparées aux données d'histoire naturelle financées par la FDA.
La FDA n'a identifié aucun problème potentiel de révision et n'a pas encore décidé de la nécessité d'une réunion du Comité Consultatif. Deramiocel a reçu la désignation de médicament orphelin de la FDA et de l'EMA, ainsi que la désignation RMAT aux États-Unis et la désignation ATMP en Europe.
Capricor Therapeutics (NASDAQ: CAPR) gab die Annahme und die Prioritätsprüfung seines Antrags auf Biologika-Lizenz (BLA) für deramiocel durch die FDA bekannt, eine Zelltherapie zur Behandlung der DMD-bedingten Kardiomyopathie. Die FDA hat einen Zieltermin für die PDUFA-Aktion auf den 31. August 2025 festgelegt.
Im Falle einer Genehmigung wäre deramiocel die erste Therapie, die speziell auf die DMD-Kardiomyopathie abzielt. Die Behandlung würde vierteljährlich als lebenslange Therapie verabreicht werden. Die Einreichung der BLA wird durch Daten aus den Phase-2-Studien HOPE-2 und HOPE-2 Open Label Extension unterstützt, die mit von der FDA finanzierten Daten zur natürlichen Krankheitsgeschichte verglichen werden.
Die FDA hat keine potenziellen Überprüfungsprobleme identifiziert und noch nicht entschieden, ob ein Treffen des Beratungsausschusses erforderlich ist. Deramiocel hat sowohl von der FDA als auch von der EMA die Orphan Drug Designation sowie die RMAT-Designierung in den USA und die ATMP-Designierung in Europa erhalten.
- FDA granted Priority Review status for deramiocel BLA
- First potential therapy for DMD cardiomyopathy if approved
- No potential review issues identified by FDA
- Multiple regulatory designations secured (Orphan Drug, RMAT, ATMP)
- Potential eligibility for Priority Review Voucher if approved by Sept 2026
- Final FDA approval still pending
- Requires lifelong quarterly treatment administration
Insights
The FDA's acceptance of Capricor's BLA for deramiocel with Priority Review is a significant regulatory milestone. This designation, which shortens the review timeline from 10 to 6 months, is only granted for therapies that may offer substantial improvements in treating serious conditions. The August 31, 2025 PDUFA date now provides a clear regulatory timeline.
Particularly encouraging is the FDA's note that no potential review issues have been identified at this time, suggesting the application package was well-prepared. Deramiocel's status as a potential first-in-class therapy for DMD cardiomyopathy addresses a critical unmet need, as currently no approved treatments exist for this specific complication which is a leading cause of death in DMD patients.
The regulatory strategy appears robust, leveraging data from Phase 2 HOPE-2 trials against natural history controls from an FDA-funded dataset. The multiple regulatory designations (Orphan Drug, RMAT, ATMP) provide advantages including enhanced FDA communication and potential market exclusivity. The potential eligibility for a Priority Review Voucher represents a valuable asset that could be monetized if approval is secured.
The quarterly administration schedule described would be favorable for long-term patient compliance, particularly important in a progressive condition requiring lifelong treatment.
This FDA acceptance with Priority Review substantially derisks Capricor's lead program and accelerates potential revenue timelines. For a company with a
The Priority Review designation not only shortens the approval timeline but also validates the unmet medical need and potential clinical value. The absence of identified review issues at this stage is atypical and reduces near-term regulatory risk, though an Advisory Committee meeting decision remains pending.
Revenue potential is enhanced by several factors: 1) Likely premium pricing as a first-in-class therapy for a fatal condition with no alternatives, 2) The quarterly administration model suggests recurring revenue, 3) Orphan designation provides 7 years of market exclusivity in the US.
The potential Priority Review Voucher represents a non-dilutive funding opportunity, as these vouchers have historically sold for
With a clear PDUFA date now established, investor attention will shift to pre-commercial activities and potential partnership opportunities as Capricor prepares for a potential Q3 2025 launch. The company's valuation will likely become increasingly tied to deramiocel's commercial prospects rather than clinical development risks.
--FDA assigns PDUFA target action date of August 31, 2025--
--If approved, deramiocel would be first therapy to treat Duchenne muscular dystrophy cardiomyopathy--
--At this time, FDA has not identified any potential review issues with the BLA--
SAN DIEGO, March 04, 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 U.S. Food and Drug Administration (“FDA”) has accepted for review its Biologics License Application (“BLA”) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (“DMD”) cardiomyopathy. Additionally, 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.
"We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies" said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape. We want to extend our appreciation to the patients, their families and advocates who continue to work with Capricor and to the FDA for its commitment to accelerating treatments for DMD.”
The BLA submission is supported by Capricor’s existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (“OLE”) trials compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The FDA also informed the Company they have not yet decided whether an Advisory Committee meeting is needed in relation to this application.
“Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,” commented Craig McDonald, M.D., national PI and University of California, Davis, Professor and Department of Physical Medicine and Rehabilitation Chair. “Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for DMD-cardiomyopathy.”
The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. 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 by September 30, 2026, Capricor would be eligible to receive a Priority Review Voucher (“PRV”) based on its previous receipt of a rare pediatric disease designation.
About Deramiocel
Deramiocel (also referred to as CAP-1002) 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
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 Capricor Therapeutics
Capricor Therapeutics, Inc. (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 demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. 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 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the Securities and Exchange Commission on November 14, 2024. 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 (CAP-1002) 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 and is not 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
FAQ
When is the FDA PDUFA date for Capricor's (CAPR) deramiocel DMD treatment?
What clinical trials support CAPR's deramiocel BLA submission?
How frequently would patients need to receive deramiocel treatment for DMD?
What regulatory designations has CAPR's deramiocel received?
What makes CAPR's deramiocel unique in the DMD treatment landscape?
