One email when the decision is public, with what it means in plain language. You'll also get new trials and FDA news for Duchenne Muscular Dystrophy. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
The FDA is expected to decide on Deramiocel for Duchenne muscular dystrophy (upper limb function) by November 22, 2026, under a BLA resubmission from Capricor Therapeutics.
What the FDA is deciding on Deramiocel for Duchenne muscular dystrophy
The FDA is reviewing Capricor Therapeutics' resubmitted application to approve Deramiocel for duchenne muscular dystrophy (upper limb function), sent back after the FDA turned down an earlier version.
Cell therapy for DMD. At a July 29, 2026 meeting, an FDA advisory committee voted 9 to 3 that the evidence did not show effectiveness for Duchenne cardiomyopathy, the use originally requested. Capricor then amended the application with 24-month open-label extension data from the Phase 3 HOPE-3 trial and asked the FDA to consider a refined indication focused on upper limb function, the trial's primary endpoint.
The FDA classified the amendment as major and extended the review 3 months, from August 22nd to November 22nd.
Who the Deramiocel decision matters to
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscle fibers from damage.
Without it, muscles deteriorate and are gradually replaced by fat and scar tissue. Multiple exon-skipping therapies (eteplirsen, golodirsen, viltolarsen, casimersen) are FDA-approved.
More about Duchenne Muscular Dystrophy
Delandistrogene moxeparvovec (Elevidys), the first gene therapy for DMD, received accelerated FDA approval in June 2023. In June 2024 the FDA granted traditional approval for ambulatory patients (those who can walk) aged 4 and older and accelerated approval for non-ambulatory patients.
After 2 deaths from acute liver failure in non-ambulatory patients, the FDA announced an investigation in June 2025, and in July 2025 it asked Sarepta to pause all shipments; on July 28, 2025 it recommended that treatment of ambulatory patients resume. In November 2025, the FDA added a boxed warning (its most serious warning) for acute serious liver injury and acute liver failure, including deaths, and limited the indication to ambulatory patients aged 4 and older, so Elevidys is no longer approved for non-ambulatory patients.
Prevalence: 1 in 3,500 to 5,000 male births. See our full Duchenne Muscular Dystrophy page for current treatments, recruiting trials, and community resources.
How Deramiocel works
Deramiocel: 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. Our full Deramiocel profile covers how it works, side effects in plain language, and its trial history.
What an approval or a rejection of Deramiocel would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. The letter describes what the FDA wants addressed, and the company then decides whether to resubmit, run more studies or stop. Either way, this page updates with the outcome and what it means.
Where Duchenne Muscular Dystrophy treatment stands today
Read our full Duchenne Muscular Dystrophy treatment summary
DMD research is remarkably active, with multiple exon-skipping therapies approved or in advanced trials, offering hope for slowing or stabilizing disease progression.
Two newer oral medicines are FDA approved for DMD regardless of the specific mutation: vamorolone (Agamree), a once-daily corticosteroid liquid approved in October 2023 for patients 2 years and older, and givinostat (Duvyzat), a twice-daily histone deacetylase (HDAC) inhibitor liquid that targets inflammation and muscle loss, approved in March 2024 for patients 6 years and older.
Gene therapy approaches are expanding, and newer antisense oligonucleotides are being tested for additional dystrophin mutations. The Muscular Dystrophy Association maintains a comprehensive trial database.
These advances are most impactful when started early, so if you have a recently diagnosed child, discuss with your neurologist which newer therapies might be appropriate and monitor emerging trial opportunities.
Genetic testing to identify your specific dystrophin mutation can help determine which targeted therapies you're eligible for.
The Duchenne Muscular Dystrophy page has every treatment and trial →Meanwhile, 79 Duchenne Muscular Dystrophy studies are open or about to open
Whatever the FDA decides here, research on Duchenne Muscular Dystrophy does not stop. The most recently updated:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Duchenne Muscular Dystrophy. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Frequently asked questions
When will the FDA decide on Deramiocel?
The FDA's target date (PDUFA date) for Deramiocel in Duchenne muscular dystrophy (upper limb function) is November 22, 2026.
Who disclosed it: Capricor Therapeutics. The FDA does not publish these dates.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
Source: Capricor press release
What is Deramiocel being reviewed for?
Capricor Therapeutics submitted a BLA resubmission for Deramiocel in Duchenne muscular dystrophy (upper limb function).
What to know: Cell therapy for DMD. At a July 29, 2026 meeting, an FDA advisory committee voted 9 to 3 that the evidence did not show effectiveness for Duchenne cardiomyopathy, the use originally requested. Capricor then amended the application with 24-month open-label extension data from the Phase 3 HOPE-3 trial and asked the FDA to consider a refined indication focused on upper limb function, the trial's primary endpoint. The FDA classified the amendment as major and extended the review 3 months, from August 22nd to November 22nd.
How it works: 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.
More: the Deramiocel drug page explains it in plain language.
Is there already an FDA-approved treatment for Duchenne Muscular Dystrophy?
Yes. Trial Friend's drug list includes 8 FDA-approved drugs for Duchenne Muscular Dystrophy.
Approved: Exondys 51 (eteplirsen), Emflaza (deflazacort), Vyondys 53 (golodirsen), Viltepso (viltolarsen), Amondys 45 (casimersen), Elevidys (delandistrogene moxeparvovec), Agamree (vamorolone), Duvyzat (givinostat).
Check: the Duchenne Muscular Dystrophy page also searches FDA drug labels for approved treatments.
What did the Deramiocel trials show?
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.
More: the Deramiocel drug page has the full trial history and sources.
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%.
More: the Deramiocel drug page has the safety details reported so far.
Can I get Deramiocel before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 79 Duchenne Muscular Dystrophy studies are open or about to open. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask Capricor Therapeutics directly. Registered programs are listed on ClinicalTrials.gov.
How much will Deramiocel cost?
Capricor Therapeutics has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the Deramiocel decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from Capricor press release. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.