Capricor Therapeutics (CAPR) has upcoming FDA regulatory milestones for Deramiocel. The table below outlines estimated target dates and event types for these pending regulatory actions.
This section highlights FDA-related milestones and regulatory updates for drugs developed by Capricor Therapeutics (CAPR).
Over the past two years, Capricor Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Deramiocel, deramiocel, StealthX, and CAP-1002. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Deramiocel FDA Regulatory Timeline and Events
Deramiocel is a drug developed by Capricor Therapeutics for the following indication: For the Treatment of Duchenne Muscular Dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Deramiocel
- Announced Date:
- August 24, 2026
- Target Action Date:
- November 22, 2026
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) target action date for its Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026.
Read Announcement- Drug:
- Deramiocel
- Announced Date:
- May 5, 2025
- Indication:
- For the Treatment of Duchenne Muscular Dystrophy
Announcement
Capricor Therapeutics announced the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company's 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.
AI Summary
Capricor Therapeutics announced the successful completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for its Biologics License Application. The application seeks full approval for deramiocel, an investigational cell therapy aimed at treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). During the meeting, the FDA confirmed that no significant deficiencies were identified in the submission.
The agency also indicated that the review process remains on track, with a Prescription Drug User Fee Act (PDUFA) target action date set for August 31, 2025, and plans to hold an advisory committee meeting in the near future. Capricor highlighted this milestone as a significant step toward providing a new treatment option that could potentially slow or halt the progression of DMD-associated cardiomyopathy.
Read Announcement
Deramiocel FDA Regulatory Timeline and Events
Deramiocel is a drug developed by Capricor Therapeutics for the following indication: For the treatment of Duchenne muscular dystrophy (DMD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- deramiocel
- Announced Date:
- July 30, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that the U.S. Food and Drug Administration's Cellular, Tissue and Gene Therapies Advisory Committee voted that available evidence did not support the effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), a rare and life-threatening disease for which no approved cardiomyopathy treatment currently exists (3 for, 9 against, 0 abstain).
AI Summary
Capricor Therapeutics announced that the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted that current evidence did not support Deramiocel’s effectiveness for treating cardiomyopathy in people with Duchenne muscular dystrophy (DMD). The vote was 3 in favor, 9 opposed, and no abstentions. DMD is a rare, life-threatening disease, and there are currently no approved treatments specifically for its cardiomyopathy.
The committee’s question covered a narrower use of Deramiocel than the indication Capricor had proposed. It also did not ask members to vote on the treatment’s overall benefit-risk profile. In a separate discussion about upper-limb function, members gave feedback that was generally supportive of clinical evidence from the Phase 3 HOPE-3 trial, including results for its primary endpoint, the PUL 2.0 measure. Deramiocel remains an investigational treatment and is not approved for commercial use.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- June 26, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced positive five-year data from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company’s lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD).
AI Summary
Capricor Therapeutics said it has positive five-year data from its ongoing HOPE-2 Open-Label Extension study of Deramiocel, its lead cell therapy candidate for Duchenne muscular dystrophy. The results suggest the treatment may help patients over a long period of time, which is important for a serious disease that weakens muscles and gets worse over time. The company said the extended study continues to support the therapy’s potential benefits in DMD.
Duchenne muscular dystrophy is a rare genetic disorder that mostly affects boys and leads to progressive loss of muscle strength. Deramiocel is designed to help protect and support damaged muscle. Capricor said the newest findings add to the growing body of evidence behind the therapy as it moves through clinical testing. The company noted that Deramiocel remains an investigational treatment and has not been approved for commercial use.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- April 22, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced the presentation of data from its Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago, Illinois.
AI Summary
Capricor Therapeutics announced it presented data from the Phase 3 HOPE‑3 clinical trial of Deramiocel for Duchenne muscular dystrophy (DMD) at the American Academy of Neurology (AAN) 2026 Annual Meeting in Chicago. The data were released under AAN embargo on April 21, 2026 at 4:45 p.m. ET. Additional meeting information is available at www.aan.com.
The company said the presentation will be posted in the Investors section of Capricor’s website shortly. Capricor emphasized that Deramiocel is an investigational therapy and has not been approved for commercial use in any indication.
Capricor also noted it has an agreement with Nippon Shinyaku Co., Ltd. (NS Pharma) for exclusive commercialization and distribution of Deramiocel in the United States and Japan, pending regulatory approval. The AAN presentation is intended to give clinicians and investors updated data on the therapy’s performance in the Phase 3 trial.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 12, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced additional analyses and new functional outcomes data from the Phase 3 HOPE-3 clinical trial of Deramiocel in Duchenne muscular dystrophy (DMD), which were presented yesterday at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Orlando, Florida.
AI Summary
Capricor Therapeutics reported additional analyses and new functional outcomes from its Phase 3 HOPE-3 trial of Deramiocel in Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando. The data, presented to clinicians and researchers, offer further detail on patient function and the trial’s endpoints, helping to clarify how Deramiocel affected measures of strength and daily activity across the study population. Capricor indicated these new analyses build on the main trial findings and provide more context for clinical interpretation.
The company said the full HOPE-3 results have been submitted for publication in a peer‑reviewed journal. The MDA presentation is available in Capricor’s Investors section, and conference details are on the MDA website. Capricor also noted a commercialization agreement with Nippon Shinyaku for the U.S. and Japan; Deramiocel remains investigational and is not approved for commercial use. Contact details are on Capricor’s site for more information.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 10, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that the U.S. Food and Drug Administration ("FDA") has lifted the previously issued Complete Response Letter and resumed review of its Biologics License Application ("BLA") seeking full approval of Deramiocel, an investigational cell therapy, for the treatment of Duchenne muscular dystrophy ("DMD") cardiomyopathy.
AI Summary
Capricor Therapeutics said the U.S. Food and Drug Administration has lifted a previously issued Complete Response Letter and resumed review of its Biologics License Application for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy. The FDA set a PDUFA target action date of August 22, 2026 and, in its response to the company, has not identified any potential review issues at this time.
Capricor noted Deramiocel has multiple regulatory designations that could speed review and confer benefits if approved, and the company expects to be eligible for a Priority Review Voucher upon approval. Deramiocel remains investigational and is not approved for commercial use.Read Announcement
- Drug:
- deramiocel
- Announced Date:
- January 20, 2026
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics today provided a regulatory update regarding its Biologics License Application (BLA) for Deramiocel, the Company's investigational first-in-class cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics provided a regulatory update on its Biologics License Application (BLA) for Deramiocel, its investigational cell therapy for Duchenne muscular dystrophy (DMD). The U.S. Food and Drug Administration has requested the full HOPE-3 clinical study report (CSR) and supporting data as part of the BLA review. The FDA did not ask for any new clinical studies or additional patient data.
Capricor says preparation of the HOPE-3 CSR is well underway and plans to submit the requested materials in February 2026. The company expects this submission to address the FDA’s questions and to support continued review of the BLA, including assignment of a new Prescription Drug User Fee Act (PDUFA) target action date. Capricor is working closely with the agency to help ensure an efficient review.
Deramiocel is a first-in-class investigational cell therapy aimed at preserving skeletal and cardiac muscle function in late-stage DMD. Capricor emphasizes that a timely review could help patients with limited treatment options.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- December 16, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics, Inc. announced that Parent Project Muscular Dystrophy (PPMD) will host a community webinar to share and discuss positive topline results from Capricor's Phase 3 HOPE-3 trial evaluating Deramiocel, the Company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics said Parent Project Muscular Dystrophy (PPMD) will host a community webinar to share and discuss positive topline results from Capricor’s Phase 3 HOPE-3 trial of Deramiocel, the company’s investigational cell therapy for Duchenne muscular dystrophy (DMD). The webinar is scheduled for Wednesday, December 17, 2025, at 1:00 p.m. ET. It will give patients and families an overview of the HOPE-3 findings and explain how those results inform ongoing regulatory discussions, including next steps with the U.S. Food and Drug Administration.
Participants are encouraged to submit questions in advance. To register or for additional details, visit Parent Project Muscular Dystrophy’s website; a replay will be posted there after the event. Deramiocel is an allogeneic cardiac-derived cell therapy in late-stage development aimed at preserving cardiac and skeletal muscle function in people with DMD. The webinar offers the community a chance to hear results directly and learn about planned regulatory and development steps.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- December 3, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced positive topline results from its pivotal Phase 3 HOPE-3 trial evaluating Deramiocel, the Company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics reported positive topline results from its pivotal Phase 3 HOPE-3 trial of Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD). The randomized, double-blind, placebo-controlled study enrolled 106 participants and met its primary endpoint: the Performance of Upper Limb (PUL v2.0) showed a 54% slowing of disease progression versus placebo (p=0.029). The key secondary cardiac endpoint, left ventricular ejection fraction (LVEF), showed a 91% slowing of decline versus placebo (p=0.041). All type 1 error-controlled secondary endpoints also achieved statistical significance, indicating consistent skeletal and cardiac benefit across measures.
Deramiocel was given intravenously (150 million cells every three months for 12 months) and was well tolerated with a safety profile consistent with earlier studies. The results support Deramiocel as a potential first-in-class therapy targeting Duchenne cardiomyopathy, with meaningful implications for preserving muscle function, protecting the heart, and improving quality of life. Detailed data are expected for scientific presentation and publication.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- September 25, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced a regulatory update for its Biologics License Application (BLA) for Deramiocel, the Company's investigational cell therapy for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics announced an FDA update for its Biologics License Application for Deramiocel, its cell therapy candidate for Duchenne muscular dystrophy. After a Type A meeting, the FDA and Capricor aligned on a clear path toward approval.
Key outcomes include using the HOPE-3 pivotal trial as the additional study requested by FDA and submitting its topline data within the current BLA to avoid a new filing and speed review.
The primary efficacy endpoint will be Performance of the Upper Limb v2.0 (PUL v2.0), with left ventricular ejection fraction (LVEF) as a key secondary measure. Capricor will seek labeling for both cardiac and skeletal muscle benefits.
HOPE-3 enrollment is complete, and topline results are expected by mid-Q4 2025 to support a BLA resubmission. Capricor believes this strategy will strengthen its clinical package and advance Deramiocel toward potential approval for DMD patients.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- September 9, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics today issued a statement regarding the U.S. Food and Drug Administration's (FDA) public posting of its Complete Response Letter (CRL) for the Biologics License Application (BLA) for Deramiocel, the Company's investigational cell therapy for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics today responded to the FDA’s unexpected public posting of its Complete Response Letter (CRL) for Deramiocel, the company’s cell therapy for cardiomyopathy in Duchenne muscular dystrophy. Although Capricor received the CRL in July 2025, it wasn’t notified that the FDA would share it publicly. The company noted that the FDA omitted Capricor’s detailed preliminary response, which clarified questions and laid out plans to address the agency’s concerns.
To keep patients and investors informed, Capricor will post its full written response on the investor section of its website. “Transparency is vital in regulatory communications,” said CEO Dr. Linda Marbán. She emphasized Capricor’s commitment to working with the FDA and giving the public a clear view of both the CRL and the company’s reply.
Capricor is now awaiting official minutes from a recent Type A meeting with the FDA, expected later this quarter. The company also looks forward to topline results from its HOPE-3 trial in the fourth quarter of 2025, which will help shape the timing of a BLA resubmission.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- August 8, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that a Type A meeting with the U.S. Food and Drug Administration (FDA) has been scheduled to discuss the regulatory path for its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics, a biotechnology firm focused on rare diseases, announced a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the regulatory pathway for its Biologics License Application (BLA) for Deramiocel. Deramiocel is the company’s lead cell therapy candidate to treat cardiomyopathy in Duchenne muscular dystrophy (DMD).
The Type A meeting will let Capricor present clinical and manufacturing data, clarify filing timelines, and align on next steps for the BLA review. Topics likely include pre-submission materials, quality documentation, and any remaining safety or efficacy questions to support approval.
To accommodate this FDA meeting, Capricor rescheduled the release of its second-quarter 2025 financial results to Monday, August 11 after market close. Management will host a live webcast and conference call at 4:30 p.m. ET that day to review financial performance and provide a corporate update.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- July 11, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics recently received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) concerning its Biologics License Application for Deramiocel. This cell therapy candidate is designed to treat cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The FDA stated that the current submission does not provide enough evidence of effectiveness and requires more clinical data. As a step forward, Capricor plans to resubmit its application with data from its ongoing Phase 3 HOPE-3 clinical trial. The company expects to have these additional findings by the third quarter of 2025. Additionally, the FDA suggested that the company should request a meeting to discuss the next steps in the approval process. Capricor remains committed to addressing the FDA’s concerns and advancing the treatment for DMD patients.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- June 24, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics today provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the Company's lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)—associated cardiomyopathy.
AI Summary
Capricor Therapeutics has provided an update on its FDA review for Deramiocel, its lead cell therapy candidate aimed at treating cardiomyopathy in Duchenne Muscular Dystrophy (DMD) patients. The FDA has clarified that an Advisory Committee meeting is not required at this time, and the Biologics License Application (BLA) remains under Priority Review. The application comes with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. Capricor expressed confidence in its submission, noting that it has successfully cleared important regulatory milestones such as the pre-license inspection and a mid-cycle review, which reported no major issues. The company is now preparing for an in-person late-cycle review meeting scheduled for mid-July and continues working closely with the FDA as it advances toward potential approval of Deramiocel.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- June 20, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD).
AI Summary
Capricor Therapeutics announced positive results from its four-year HOPE-2 Open-Label Extension study of Deramiocel, a cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The study shows that patients treated with Deramiocel experienced a median change of –0.5 points compared to baseline, highlighting the treatment’s ability to slow disease progression. A subgroup of patients with a baseline left ventricular ejection fraction (LVEF) greater than 45% saw even stronger benefits, suggesting that early treatment may help preserve cardiac function. In addition, measurements with the Performance of the Upper Limb (PUL v2.0) tool indicated a smaller decline in skeletal muscle function in the fourth year (0.6 points) compared to the first year (1.8 points). Throughout the study, Deramiocel maintained a favorable safety profile, supporting its potential as a long-term treatment option for DMD patients.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 17, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced positive long-term data from its ongoing HOPE-2 open label extension ("OLE") clinical trial, demonstrating the potential of the Company's lead asset, deramiocel, to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy ("DMD").
AI Summary
Capricor Therapeutics announced encouraging long-term results from its ongoing HOPE-2 open label extension clinical trial. The study shows that patients with Duchenne muscular dystrophy (DMD) treated with deramiocel experienced a slower decline in upper limb function compared to a matched external group. Over three years, the deramiocel-treated group had an average performance decline of 3.46 points versus 7.19 points in the comparison group—a 52% reduction in disease progression.
This data suggests that deramiocel may help slow DMD progression and preserve muscle function over time. The trial also showed that the treatment effect increased each year and that deramiocel was well tolerated with no new safety concerns. Overall, these findings provide hope for potentially transforming life quality for patients affected by DMD.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 10, 2025
- Target Action Date:
- August 31, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics, Inc announced that The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") target action date of August 31, 2025
AI Summary
Capricor Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for deramiocel, an investigational cell therapy designed to treat Duchenne muscular dystrophy cardiomyopathy. The FDA granted the BLA Priority Review along with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025. This accelerated review process is intended to speed up the development and potential approval of therapies that address serious conditions with limited treatment options.
The Priority Review status means that the agency will work to complete its evaluation of the therapy within a shortened timeframe. Capricor Therapeutics’ commitment to advancing deramiocel brings additional hope for patients with Duchenne muscular dystrophy, as this condition greatly impacts the heart and overall quality of life. The news is a significant step forward in the development of innovative therapies for rare diseases.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 10, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
NS Pharma, Inc. announced that acceptance has been received by Capricor Therapeutics, Inc 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.
AI Summary
NS Pharma, Inc. announced that Capricor Therapeutics, Inc. has received acceptance from the U.S. Food and Drug Administration (FDA) for its Biologics License Application (BLA) filing for deramiocel. Deramiocel is an investigational cell therapy intended to treat cardiomyopathy in patients diagnosed with Duchenne muscular dystrophy (DMD). The FDA has granted this filing a Priority Review, with a targeted decision date of August 31, 2025. This treatment uses allogeneic cardiosphere-derived cells, which have shown promise in reducing inflammation and promoting healing in the heart. NS Pharma will be responsible for the commercialization and distribution of deramiocel in the United States. The companies involved believe that if approved, this innovative therapy could help fill a key gap in treatment options for patients suffering from DMD-related heart complications.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 4, 2025
- Target Action Date:
- August 31, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that , 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.
AI Summary
Capricor Therapeutics announced that the FDA has granted its Biologics License Application (BLA) for deramiocel a Priority Review, with a Prescription Drug User Fee Act target action date set for August 31, 2025. The agency confirmed that, at this time, there are no identified potential review issues with the application.
If approved, deramiocel is expected to become the first therapy aimed specifically at treating cardiomyopathy in patients with Duchenne muscular dystrophy. This condition is a leading cause of death in those with DMD, and the approval would mark a significant milestone in addressing a serious unmet medical need. The decision to grant Priority Review underscores the FDA’s commitment to expediting treatments that offer meaningful improvements in safety or effectiveness for severe conditions.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- March 4, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics 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.
AI Summary
Capricor Therapeutics announced that the FDA has accepted its Biologics License Application for deramiocel, an investigational cell therapy aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy. The agency granted the application Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025, and has not identified any potential review issues at this stage. If approved, deramiocel would be the first-ever therapy to treat DMD cardiomyopathy, offering hope to patients who currently have limited treatment options. Capricor’s submission is supported by existing Phase 2 clinical data and highlights deramiocel’s potential to provide significant cardiac benefits. The company views this regulatory milestone as a crucial step toward delivering a first-in-class, potentially lifelong treatment administered quarterly to improve both the safety and effectiveness of care for those suffering from this serious condition.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- January 2, 2025
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics y announced the completion of the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel, an investigational cell therapy, to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
AI Summary
Capricor Therapeutics announced the completion of its Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for deramiocel, an investigational cell therapy designed to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. This therapy, if approved, would become the first approved treatment for the heart complications caused by DMD, addressing a significant unmet need in patient care.
The submission represents years of research and positive clinical trial results, where deramiocel has shown potential in lessening cardiac issues associated with DMD. Along with advancing this novel treatment option, Capricor will also receive a $10 million milestone payment from its partner Nippon Shinyaku. The company has requested a priority review, aiming to shorten the FDA review timeline and bring this transformative therapy closer to patients in need.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- October 11, 2024
- Target Action Date:
- LATE 2024
- Estimated Target Date Range:
- October 1, 2024 - December 31, 2024
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced that Company on Track to Fully Submit Biologics License Application (BLA) by End of 2024-
AI Summary
Capricor Therapeutics announced that it is on track to fully submit its Biologics License Application (BLA) to the FDA by the end of 2024. This submission will include key data from a three-year study called HOPE-2, which examined the safety and effectiveness of its lead product, deramiocel, for treating Duchenne muscular dystrophy (DMD). The study shows that deramiocel provides long-term benefits to both heart and skeletal muscles, an important finding since heart complications are a major cause of death in DMD patients.
The positive results highlight the potential of deramiocel to protect heart function and improve muscle strength over time. Capricor’s leadership emphasized that early and continuous treatment can help preserve heart function, which is critical because lost function is hard to recover. The upcoming BLA submission is a significant step toward making this promising therapy available to patients battling DMD.
Read Announcement- Drug:
- deramiocel
- Announced Date:
- October 11, 2024
- Indication:
- For the treatment of Duchenne muscular dystrophy (DMD)
Announcement
Capricor Therapeutics announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS), October 8-12, 2024 in Prague, Czechia.
AI Summary
Capricor Therapeutics announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension study for deramiocel, its lead treatment for Duchenne muscular dystrophy (DMD). The study showed that continuous treatment with deramiocel led to sustained improvements in both cardiac and skeletal muscle functions, with significant benefits observed in multiple cardiac endpoints such as left ventricular ejection fraction and other measures. Additionally, improvements in skeletal muscle function were demonstrated through enhanced PUL v2.0 scores over the three-year period. These encouraging results were presented in a late-breaking poster session at the 29th Annual Congress of the World Muscle Society (WMS) in Prague, Czechia, highlighting deramiocel’s potential as a transformative lifelong therapy for DMD patients, particularly in addressing DMD-associated cardiomyopathy. The favorable safety and efficacy profile supports the company’s plan to fully submit its Biologics License Application (BLA) by the end of 2024.
Read Announcement
StealthX FDA Regulatory Events
StealthX is a drug developed by Capricor Therapeutics for the following indication: exosome-based vaccine.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- StealthX
- Announced Date:
- August 18, 2025
- Indication:
- exosome-based vaccine.
Announcement
Capricor Therapeutics announced that the first subjects have been dosed in a Phase 1 clinical trial evaluating its StealthX™ exosome-based vaccine.
AI Summary
Capricor Therapeutics announced that it has dosed the first participants in a Phase 1 clinical trial of its StealthX™ exosome-based vaccine. The study is funded by the National Institute of Allergy and Infectious Diseases under the U.S. Department of Health and Human Services’ Project NextGen.
The trial includes four dosing arms initially focused on the SARS-CoV-2 spike protein, with a planned arm incorporating the nucleocapsid protein pending additional clearance. Capricor expects to report initial safety and immune response data in the first quarter of 2026.
StealthX™ uses engineered exosomes to display viral proteins without adjuvants, offering a natural delivery system that relies on native proteins. This design supports rapid adaptation to emerging targets and may reduce side effects compared with traditional platforms.
With first-in-human dosing achieved, Capricor aims to advance StealthX™ as a versatile platform for multivalent vaccines and targeted therapies, potentially driving future partnerships and pipeline expansion.
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CAP-1002 FDA Regulatory Events
CAP-1002 is a drug developed by Capricor Therapeutics for the following indication: Duchenne muscular dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- CAP-1002
- Announced Date:
- October 9, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced today that it has initiated its rolling submission process with the U.S. Food and Drug Administration (FDA) for a Biologics License Application (BLA), seeking full approval for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
AI Summary
Capricor Therapeutics announced that it has started a rolling submission with the U.S. Food and Drug Administration (FDA) for its Biologics License Application (BLA). The application seeks full FDA approval for deramiocel as a treatment for Duchenne muscular dystrophy (DMD) cardiomyopathy, a serious heart condition affecting all DMD patients. This submission is an important step in getting a novel, first-in-class therapy to the patients who need it, as there are currently no approved treatment options for the cardiac complications of DMD. Capricor plans to complete the rolling submission by the end of 2024, and the application might be eligible for priority review if it shows significant improvements in safety and effectiveness. The company is eager to work closely with the FDA throughout this process to help expedite the potential approval and delivery of deramiocel.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- October 4, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics
AI Summary
Capricor Therapeutics is set to reveal important three-year safety and efficacy results from its HOPE-2 open-label extension study for Duchenne muscular dystrophy (DMD). The company will present data at the 29th Annual Congress of the World Muscle Society in Prague, highlighting the long-term and multi-modal benefits of deramiocel (CAP-1002). This investigational cell therapy has shown promising effects on both skeletal and cardiac muscles, offering a potential new treatment for patients with DMD-cardiomyopathy.
Linda Marbán, Ph.D., CEO of Capricor, emphasized that these results support the company’s plans to file a Biologics License Application. This filing could lead to a significant step forward in securing approval for deramiocel, marking an important milestone for patients with DMD. The study’s results underline the ongoing promise and potential impact of Capricor’s innovative treatment approach.
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