Capricor Therapeutics Granted Orphan Drug and ATMP Status for Deramiocel by European Medicines Agency
Capricor Therapeutics (NASDAQ: CAPR) has received both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations from the European Medicines Agency for deramiocel, its lead asset targeting Duchenne muscular dystrophy (DMD). The Orphan Drug status grants 10-year market exclusivity upon approval and reduced regulatory fees, while ATMP designation provides regulatory support for cell-based therapy development. The company has also initiated a rolling Biologics License Application with the FDA, expecting completion by end of 2024, seeking full approval for deramiocel in treating DMD-cardiomyopathy.
Capricor Therapeutics (NASDAQ: CAPR) ha ricevuto le designazioni di Farmaco Orfano e Prodotto Medicinale per Terapia Avanzata (ATMP) dall'Agenzia Europea per i Medicinali per deramiocel, il suo principale asset destinato alla distrofia muscolare di Duchenne (DMD). Lo status di Farmaco Orfano garantisce un'esclusiva di mercato di 10 anni al momento dell'approvazione e riduzioni delle tasse regolatorie, mentre la designazione ATMP fornisce supporto normativo per lo sviluppo di terapie cellulari. L'azienda ha anche avviato una richiesta di Licenza Biologica presso la FDA, prevedendo il completamento entro la fine del 2024, cercando l'approvazione completa per deramiocel nel trattamento della DMD-cardiomiopatia.
Capricor Therapeutics (NASDAQ: CAPR) ha recibido ambas designaciones de Medicamento Huérfano y Producto Medicinal de Terapia Avanzada (ATMP) de la Agencia Europea de Medicamentos para deramiocel, su activo principal dirigido a la distrofia muscular de Duchenne (DMD). El estado de Medicamento Huérfano otorga 10 años de exclusividad en el mercado tras la aprobación y tarifas regulatorias reducidas, mientras que la designación ATMP proporciona apoyo regulatorio para el desarrollo de terapias celulares. La empresa también ha iniciado una Solicitud de Licencia Biológica continua con la FDA, esperando completarla a finales de 2024, buscando la aprobación total para deramiocel en el tratamiento de la DMD-cardiomiopatía.
Capricor Therapeutics (NASDAQ: CAPR)는 유럽 의약품 청으로부터 뒤셴 근육형성증(DMD)을 목표로 하는 주요 자산인 deramiocel에 대해 희귀의약품 및 고도치료의약품(ATMP) 지정을 받았습니다. 희귀의약품 status는 승인 후 10년간의 시장 독점권과 규제 수수료 인하를 부여하고, ATMP 지정은 세포 기반 치료 개발을 위한 규제 지원을 제공합니다. 이 회사는 또한 FDA와 함께 계속적인 생물학적 라이센스 신청을 시작하였으며, 2024년 말까지 완료될 것으로 기대하고 있으며, DMD-심장병 치료를 위한 deramiocel의 전체 승인을 추구하고 있습니다.
Capricor Therapeutics (NASDAQ: CAPR) a reçu à la fois les désignations de Médicament Orphelin et de Produit Médical de Thérapie Avancée (ATMP) de l'Agence Européenne des Médicaments pour deramiocel, son principal actif visant la dystrophie musculaire de Duchenne (DMD). Le statut de Médicament Orphelin accorde une exclusivité de marché de 10 ans après approbation et réduit les frais réglementaires, tandis que la désignation ATMP fournit un soutien réglementaire pour le développement de thérapies cellulaires. L'entreprise a également lancé une demande de Licence Biologique continue auprès de la FDA, s'attendant à la finaliser d'ici la fin de 2024, cherchant l'approbation complète pour deramiocel dans le traitement de la DMD-cardiomyopathie.
Capricor Therapeutics (NASDAQ: CAPR) hat von der Europäischen Arzneimittel-Agentur sowohl die Auszeichnung als Waisenarzneimittel als auch als Arzneimittel zur fortgeschrittenen Therapie (ATMP) für deramiocel, sein Hauptprodukt zur Behandlung der Duchenne-Muskeldystrophie (DMD), erhalten. Der Status als Waisenarzneimittel gewährt 10 Jahre Marktexklusivität nach der Genehmigung sowie reduzierte regulatorische Gebühren, während die ATMP-Auszeichnung regulatorische Unterstützung für die Entwicklung von zellbasierten Therapien bietet. Das Unternehmen hat außerdem einen fortlaufenden Antrag auf biologisches Lizenzverfahren bei der FDA eingereicht, dessen Abschluss bis Ende 2024 erwartet wird, um die vollständige Genehmigung für deramiocel zur Behandlung der DMD-Kardiomyopathie zu erlangen.
- Received EMA Orphan Drug designation granting 10-year market exclusivity
- Obtained ATMP designation providing regulatory support and potential faster development
- Initiated rolling BLA submission with FDA for full approval
- Secured potential market exclusivity in both US and European markets
- None.
Insights
The dual EMA designations significantly strengthen Capricor's market position for deramiocel in Europe. The 10-year market exclusivity from Orphan Drug status provides important competitive protection, while ATMP designation offers valuable regulatory support and potential acceleration of development timelines. These designations, combined with the ongoing BLA submission in the US, create a robust regulatory framework across major markets.
The regulatory milestones enhance deramiocel's commercial potential in treating DMD, a rare disease with significant unmet needs. The expedited pathways and reduced fees will help optimize resource allocation during the development phase. With both US and EU regulatory support now secured, Capricor is well-positioned to potentially capture significant market share in the DMD treatment space if deramiocel receives approval.
These regulatory designations significantly de-risk Capricor's path to commercialization in Europe. The reduced regulatory fees and development support will help conserve capital, while the potential market exclusivity creates a clear revenue opportunity. The concurrent US BLA submission indicates strong execution of a global commercialization strategy.
The DMD market represents a valuable opportunity due to high unmet need and potential pricing power for orphan drugs. With a comprehensive regulatory strategy now in place across major markets, Capricor is strengthening its competitive position. The company's focus on completing the BLA submission by end of 2024 provides a clear near-term catalyst for potential value creation.
Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular dystrophy in the European region
SAN DIEGO, Nov. 20, 2024 (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 European Medicines Agency (EMA) has granted both Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations to its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD).
The Orphan Drug designation provides Capricor with several benefits that support the development of deramiocel in Europe, including market exclusivity for 10 years if approval is granted and substantially reduced regulatory fees. The ATMP designation provides substantial regulatory support to assist in the development of cell-based therapies. The additional support can potentially reduce time to market, streamline development, and open up access to critical resources, making it an invaluable tool for companies working on innovative therapies.
“Receiving the Orphan Drug and ATMP designations from the EMA are significant steps forward as we work to bring deramiocel to DMD patients worldwide,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “With these designations from the EMA, along with Orphan Drug and RMAT designations from the U.S Food and Drug Administration (FDA), we have the opportunity to secure market exclusivity for deramiocel in two of the world’s key markets. These milestones reflect our commitment to advancing novel, transformative treatments for patients suffering from DMD.”
Recently, Capricor announced that it had initiated its rolling Biologics License Application (BLA) submission process with the U.S. FDA seeking full approval of deramiocel to treat all patients diagnosed with DMD-cardiomyopathy. Capricor expects to complete its full BLA submission by the end of 2024.
About Deramiocel
Deramiocel (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. Deramiocel for the treatment of DMD has previously received Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation (RMAT) from the U.S. FDA. In addition, if deramiocel is approved, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on our previous receipt of a rare pediatric disease designation.
About Duchenne Muscular Dystrophy
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 (CAP-1002), 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 advancing through Phase 3 clinical 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
What designations did Capricor Therapeutics (CAPR) receive from EMA for deramiocel?
When does Capricor Therapeutics (CAPR) expect to complete its BLA submission for deramiocel?
What market exclusivity period will CAPR receive in Europe for deramiocel if approved?
