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
CAP-1002, deramiocel, Deramiocel, and StealthX. 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.
CAP-1002 FDA Regulatory Timeline and 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.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- June 25, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA (Biologics License Application) meeting with the Company in the third quarter of 2024 for deramiocel (CAP-1002), for the treatment of Duchenne Muscular Dystrophy (DMD).
AI Summary
Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has scheduled a Pre-BLA meeting in the third quarter of 2024 for its product deramiocel (CAP-1002), which is aimed at treating Duchenne Muscular Dystrophy (DMD). This meeting is a critical step as Capricor plans to finalize its Biologics License Application (BLA) filing plans and explore options to expedite the submission.
The company’s goal is to use all available data to guide their filing process and set a rolling timeline with the FDA. The scheduled meeting reflects the ongoing efforts at Capricor to move closer to potential approval of deramiocel, with an emphasis on addressing the many challenges faced by DMD patients. The discussions with the FDA will play a key role in the future development of this innovative therapy.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- June 11, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced the successful completion of a Type-B meeting with the U.S. Food and Drug Administration (FDA) on next steps for the Biologics License Application (BLA) submission with its lead asset, CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics recently announced that it successfully completed a Type-B meeting with the U.S. Food and Drug Administration. The meeting focused on the next steps for its Biologics License Application submission for CAP-1002, the company’s lead asset aimed at treating Duchenne muscular dystrophy. During the meeting, the FDA agreed to a pre-BLA discussion based on the review of CAP-1002’s clinical data. This key decision allows Capricor to start a rolling BLA submission, with the process planned to begin in the third quarter of 2024. The rolling submission method lets the company file finished parts of the application as they are completed, potentially accelerating the overall review and reducing risks. Capricor’s progress in this phase marks an important step toward securing approval for CAP-1002 and moving closer to the commercialization of the treatment.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- June 4, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced positive 3-year results from the ongoing HOPE-2 open-label extension (OLE) study with CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Capricor Therapeutics announced positive three-year results from the ongoing HOPE-2 open-label extension (OLE) study using CAP-1002 for Duchenne muscular dystrophy (DMD). The study showed that CAP-1002 treatment led to significant benefits in skeletal muscle function, as measured by the PUL 2.0 test (p<0.001), when compared to an external group of similar DMD patients. These results suggest that the drug may slow the decline in upper limb function commonly seen in DMD.
Additionally, patients experienced stabilization of cardiac function, with left ventricular ejection fraction (LVEF) remaining steady over the three-year period. CAP-1002 was well tolerated by participants throughout the study, supporting its potential as a long-term treatment option for those suffering from this progressive and debilitating disease.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- April 24, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced that
AI Summary
Capricor Therapeutics announced that the FDA has affirmed that the company has successfully demonstrated non-clinical comparability between drug products made at its Los Angeles and San Diego facilities. This endorsement means that following potential approval, Capricor can immediately use its San Diego manufacturing facility for CAP-1002. The agency also advised the company to include discussions about a pre-BLA meeting and a rolling BLA submission on its agenda. In response, Capricor has been granted another Type-B meeting scheduled for the second quarter of 2024 to further discuss these strategies. This feedback is important for Capricor as it continues to advance CAP-1002, a lead asset for treating Duchenne muscular dystrophy. The company will share more updates on its progress during a virtual investor webcast on April 29, aiming to expedite its path toward potential regulatory approval.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- April 24, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced an update from the Company's recent Type-B Chemistry, Manufacturing and Controls ("CMC") meeting with the U.S. Food and Drug Administration ("FDA") on next steps for the Biologics License Application ("BLA") submission with its lead asset CAP-1002 in treating Duchenne muscular dystrophy ("DMD").
AI Summary
Capricor Therapeutics recently updated its stakeholders on a Type-B Chemistry, Manufacturing and Controls (CMC) meeting with the FDA regarding the next steps for the Biologics License Application (BLA) for its lead asset, CAP-1002, in treating Duchenne muscular dystrophy (DMD). During the meeting, the FDA supported discussions on holding a pre-BLA meeting and implementing a rolling BLA submission, with a follow-up meeting scheduled for the second quarter of 2024. This feedback aims to potentially accelerate the approval process for CAP-1002.
The FDA also confirmed that the analytical data show comparability between CAP-1002 drug products manufactured at the Los Angeles and San Diego facilities. This established non-clinical comparability allows the San Diego plant to start operations upon potential product approval, letting the company focus resources on readiness for launch as it collaborates further with the FDA.
Read Announcement- Drug:
- CAP-1002
- Announced Date:
- April 24, 2024
- Indication:
- Duchenne muscular dystrophy
Announcement
Capricor Therapeutics announced that Company Granted Subsequent Type-B Clinical Meeting with FDA in Second Quarter to Continue to Discuss Pathway to BLA-
AI Summary
Capricor Therapeutics has been granted a subsequent Type-B clinical meeting with the FDA in the second quarter of 2024. The company will use this meeting to focus on discussing the pathway to a Biologics License Application (BLA) for its lead product, CAP-1002, aimed at treating Duchenne muscular dystrophy. During the session, Capricor plans to talk specifically about its request for a pre-BLA meeting and the strategy for a rolling BLA submission to potentially accelerate the filing process. The FDA’s support in these discussions is seen as a positive step towards streamlining the regulatory review. This upcoming meeting is a crucial step in expediting CAP-1002's approval process and moving closer to delivering new treatment options for patients with rare diseases.
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:
- 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
- Estimated Event Date Range:
- August 31, 2025 - August 31, 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
- 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:
- March 4, 2025
- Estimated Event Date Range:
- August 31, 2025 - August 31, 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:
- 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
- Estimated Event Date Range:
- October 1, 2024 - December 31, 2024
- Target Action Date:
- LATE 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
Deramiocel FDA Regulatory 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:
- 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.
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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.
Read Announcement
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