Deramiocel
An investigational treatment for Duchenne Muscular Dystrophy.
The same compound appears under different names depending on the context. Here is how to identify Deramiocel wherever you encounter it, plus the key facts at a glance.
- Generic name
- Deramiocel
- Development code
- CAP-1002
- Drug class
- Allogeneic cell therapy
- Manufacturer
- Capricor Therapeutics
- How it's taken
- An IV infusion of 150 million cells every 3 months, the dose used in HOPE-3.
Capricor Therapeutics' investigational cell therapy for Duchenne muscular dystrophy, made from heart cells grown from donated tissue and given by IV infusion every 3 months. The FDA turned down Capricor's first application in July 2025, an FDA advisory committee voted 9 to 3 against it for Duchenne cardiomyopathy in July 2026, and the FDA is now reviewing Capricor's request for an upper limb indication, with a decision due November 22, 2026.
The decision is expected by November 22, 2026. One email when it is public, with what it means in plain language, plus new trials for Duchenne Muscular Dystrophy and FDA news. Unsubscribe anytime.
You'll get new recruiting trials, changes to trials, FDA decisions and condition news for Duchenne Muscular Dystrophy. Turn any of these off from the link in every email.
Where Deramiocel fits
If approved, deramiocel would be the first cell therapy for Duchenne. Most HOPE-3 participants no longer walked, a stage where few treatments have been tested, and all of them stayed on steroids, so it would be added to steroid treatment rather than replace it. Its approved use, if any, depends on which indication the FDA accepts: cardiomyopathy, which the advisory committee voted against, or upper limb function, which Capricor is now requesting.
How Deramiocel works
Deramiocel is made of cardiosphere-derived cells, a type of cell grown from donated human heart tissue. Because the cells come from a donor rather than from the patient, one manufactured batch can treat many people. Capricor says the cells work mainly by releasing tiny packets called exosomes, which reach immune cells called macrophages and shift them from an inflammatory state toward one that supports healing, with the aim of slowing the inflammation and scarring that destroy heart and skeletal muscle in Duchenne. Deramiocel does not restore dystrophin, the missing protein that causes Duchenne. In HOPE-3, every participant stayed on a stable steroid regimen.
Mechanism: Donor heart-derived cells (cardiosphere-derived cells); Capricor proposes that they release exosomes that shift immune cells called macrophages from an inflammatory state toward a healing one
Side effects and safety
The side effect that stands out is allergic-type reactions linked to the infusion. In HOPE-3, the FDA's briefing document counted hypersensitivity reactions in 22 of 53 participants on deramiocel, about 42%, versus 8 of 52 on placebo, about 15%.
In the earlier HOPE-2 trial, 3 of 8 treated participants had hypersensitivity reactions. One had a severe allergic reaction during his second infusion that needed emergency treatment and a hospital stay, and he stopped treatment. HOPE-2 then added steroid and antihistamine medicines before each infusion, and reactions became less common.
Capricor reports giving about 1,300 infusions to more than 200 people with Duchenne across 3 trials and describes the long-term safety profile as consistent. There is no FDA label yet, so the full list of warnings is not known.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Deramiocel
An IV infusion of 150 million cells every 3 months, the dose used in HOPE-3. After an early severe reaction, HOPE-2 began giving steroid and antihistamine medicines before each infusion to lower the risk of an allergic reaction. If the FDA approves deramiocel, its label will set the final dose, schedule and premedication.
Clinical trial results
HOPE-3 (NCT05126758) randomly assigned 106 boys and young men aged 10 to 22, most no longer able to walk, at 20 U.S. sites to deramiocel or placebo every 3 months for 12 months.
Capricor reported that deramiocel slowed decline on the PUL 2.0 scale of arm and hand function by 54% versus placebo (p=0.029), and The Lancet published the results in July 2026. During peer review Capricor corrected its heart pumping result, and the difference in left ventricular ejection fraction became 1.8 percentage points (p=0.09), down from 2.4 points (p=0.04) in the topline report.
The FDA's briefing document said HOPE-3 did not meet its pre-specified endpoints, that the analysis plan was changed after the double-blind period ended, and that the uneven rate of allergic reactions could have let some people guess who was getting the drug. Capricor says its final plan, version 3.0, was locked before the data were unblinded, and The Lancet published the results under that plan.
In the open-label extension, which 98 participants entered, Capricor reported that people who switched from placebo had a 76% smaller decline in arm function in their first treated year than in their placebo year, an exploratory before-and-after comparison that the study was not designed or powered to test.
HOPE-2 (NCT03406780), a Phase 2 trial that stopped at 20 participants, reported slower upper limb decline in The Lancet in 2022. The FDA's briefing document said it showed no significant effect on its primary or secondary endpoints.
Main registered trial: NCT05126758 on ClinicalTrials.gov. Check it for the current status, sites and contacts before asking about enrollment.
Development history
Capricor first tested the cells in heart attack survivors, then turned to the heart and muscle damage of Duchenne with HOPE-Duchenne and HOPE-2.
It completed a biologics license application at the end of December 2024 seeking approval for Duchenne cardiomyopathy, and the FDA accepted it with priority review in March 2025. On July 11, 2025 Capricor announced a complete response letter saying the application did not meet the requirement for substantial evidence of effectiveness, called for more clinical data and cited outstanding manufacturing items.
After HOPE-3's topline results in December 2025, the FDA accepted Capricor's response as a Class 2 resubmission on March 10, 2026, with a goal date of August 22, 2026.
On July 29, 2026 the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9 to 3 that the evidence did not show effectiveness for cardiomyopathy. It was not asked to vote on the upper limb result.
Capricor then amended the application with 24-month data and a refined indication focused on upper limb function. On August 24, 2026 it said the FDA had classified the amendment as major and moved the goal date to November 22, 2026. Deramiocel also holds FDA orphan drug designation for Becker muscular dystrophy, granted in June 2025.
Explore Duchenne Muscular Dystrophy trials
Other Duchenne Muscular Dystrophy treatments
Deramiocel in Trial Friend News and Guides
Common questions about Deramiocel
▸What is Deramiocel?
Capricor Therapeutics' investigational cell therapy for Duchenne muscular dystrophy, made from heart cells grown from donated tissue and given by IV infusion every 3 months. The FDA turned down Capricor's first application in July 2025, an FDA advisory committee voted 9 to 3 against it for Duchenne cardiomyopathy in July 2026, and the FDA is now reviewing Capricor's request for an upper limb indication, with a decision due November 22, 2026.
▸How does Deramiocel work?
Deramiocel is made of cardiosphere-derived cells, a type of cell grown from donated human heart tissue. Because the cells come from a donor rather than from the patient, one manufactured batch can treat many people. Capricor says the cells work mainly by releasing tiny packets called exosomes, which reach immune cells called macrophages and shift them from an inflammatory state toward one that supports healing, with the aim of slowing the inflammation and scarring that destroy heart and skeletal muscle in Duchenne. Deramiocel does not restore dystrophin, the missing protein that causes Duchenne. In HOPE-3, every participant stayed on a stable steroid regimen.
▸What are the side effects of Deramiocel?
The side effect that stands out is allergic-type reactions linked to the infusion. In HOPE-3, the FDA's briefing document counted hypersensitivity reactions in 22 of 53 participants on deramiocel, about 42%, versus 8 of 52 on placebo, about 15%.
In the earlier HOPE-2 trial, 3 of 8 treated participants had hypersensitivity reactions. One had a severe allergic reaction during his second infusion that needed emergency treatment and a hospital stay, and he stopped treatment. HOPE-2 then added steroid and antihistamine medicines before each infusion, and reactions became less common.
Capricor reports giving about 1,300 infusions to more than 200 people with Duchenne across 3 trials and describes the long-term safety profile as consistent. There is no FDA label yet, so the full list of warnings is not known.
▸How is Deramiocel taken?
An IV infusion of 150 million cells every 3 months, the dose used in HOPE-3. After an early severe reaction, HOPE-2 began giving steroid and antihistamine medicines before each infusion to lower the risk of an allergic reaction. If the FDA approves deramiocel, its label will set the final dose, schedule and premedication.
▸Is Deramiocel FDA approved?
Not yet. Deramiocel is under FDA review for Duchenne Muscular Dystrophy; Capricor Therapeutics has filed an application and a decision is due November 22, 2026. The FDA calendar page tracks the outcome.
▸When will the FDA decide on deramiocel?
The FDA's goal date is November 22, 2026. It was August 22, 2026 until Capricor sent 24-month HOPE-3 data and asked the FDA to consider an indication focused on upper limb function; the FDA classified that as a major amendment and added 3 months. The FDA can act before the goal date, and the FDA calendar page for deramiocel will show the outcome the day it is public.
▸Is deramiocel FDA approved?
No. Deramiocel is not approved for any use. The FDA turned down Capricor's first application, for Duchenne cardiomyopathy, in July 2025, saying it lacked substantial evidence of effectiveness. Capricor resubmitted with Phase 3 HOPE-3 data, and the FDA is due to decide by November 22, 2026.
▸What did the FDA advisory committee vote on deramiocel?
On July 29, 2026 the FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9 to 3 that the evidence did not show deramiocel is effective for cardiomyopathy in Duchenne, the use Capricor originally requested. The committee was not asked to vote on HOPE-3's upper limb result or on overall benefit and risk, and Capricor describes its separate discussion of upper limb function as directionally supportive. The vote is advice; the FDA makes the decision.
▸Can my son get deramiocel now?
Only people already in Capricor's studies receive it today. HOPE-3 and the HOPE-2 open-label extension are listed on ClinicalTrials.gov as active but not recruiting, and we could not find an announced expanded access program. If the FDA approves deramiocel, its label will say who can receive it. Ask your son's neuromuscular team about recruiting Duchenne trials, which are listed on our Duchenne page.
▸What are the side effects of deramiocel?
Allergic-type reactions linked to the infusion are the main concern. In HOPE-3 the FDA counted hypersensitivity reactions in about 42% of participants on deramiocel versus about 15% on placebo. In HOPE-2, 1 participant had a severe allergic reaction that needed emergency treatment and a hospital stay, and reactions became less common after steroid and antihistamine premedication was added. Capricor describes its long-term safety experience across about 1,300 infusions as consistent.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Capricor Therapeutics · 2026-10-05. Capricor Therapeutics Presents Positive HOPE-3 Open-Label Extension Data on Upper Limb Function in Duchenne Muscular Dystrophy at 2026 World Muscle Society Congress. https://www.capricor.com/investors/news-events/press-releases/detail/356/capricor-therapeutics-presents-positive-hope-3-open-label
- Capricor Therapeutics · 2026-08-24. Capricor Therapeutics Announces Extension of PDUFA Target Action Date as FDA Continues Review of Deramiocel BLA. https://www.capricor.com/investors/news-events/press-releases/detail/354/capricor-therapeutics-announces-extension-of-pdufa-target
- Capricor Therapeutics · 2026-08-13. Capricor Therapeutics Reports Second Quarter 2026 Financial Results and Provides Corporate Update. https://www.capricor.com/investors/news-events/press-releases/detail/353/capricor-therapeutics-reports-second-quarter-2026-financial
- Capricor Therapeutics · 2026-07-30. Capricor Therapeutics Provides Update on FDA Advisory Committee Meeting for Deramiocel. https://www.capricor.com/investors/news-events/press-releases/detail/351/capricor-therapeutics-provides-update-on-fda-advisory
- U.S. Food and Drug Administration · 2026-07. FDA briefing document: Cellular, Tissue, and Gene Therapies Advisory Committee meeting on deramiocel. https://www.fda.gov/media/193839/download
- The Lancet · 2026-07. Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01385-1/fulltext
- Capricor Therapeutics · 2025-07-11. Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy. https://www.capricor.com/investors/news-events/press-releases/detail/319/capricor-therapeutics-provides-regulatory-update-on
- The Lancet · 2022-03. Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. https://doi.org/10.1016/S0140-6736(22)00012-5
- ClinicalTrials.gov. A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3). https://clinicaltrials.gov/study/NCT05126758