Registered forecast
35% registered probability event by 2026-11-22 Unresolved
FDA approves deramiocel for Duchenne muscular dystrophy on or before the disclosed PDUFA goal date of 2026-11-22
FF-008 Registered - Registered probability
- 35%
- Event by
- 2026-11-22
- Resolution window
- 2026-09-27 → 2026-11-22
- Target rung
- 5 Guideline
- 6 Regulatory
- 7 Coverage
- Baselines it has to beat
-
- Authority base rate 50%
- Calendar cycle 60%
- Analyst proxy 35%
nextconsensus.com/forecast/ff-008/ registry snapshot 2026-10-06 Share image
NextConsensus registered this probability on 2026-09-26. It is one of 14 registered FDA goal-date forecasts.
The proposition
What was claimed
The FDA takes an approval action on Capricor Therapeutics' Biologics License Application for deramiocel (CAP-1002) for the treatment of Duchenne muscular dystrophy, with an approval action date on or before 2026-11-22, the PDUFA target action date disclosed in Capricor's Form 8-K filed 2026-08-24.
- Authority
- US Food and Drug Administration (FDA), Center for Biologics Evaluation and Research (CBER) (US)
- Target rungA step on the 12-rung ladder: rungs 1 to 4 are the evidence moving, rungs 5 to 7 are a guideline body, regulator, or payer acting. The ladder, rung by rung
- Rung 6: Regulatory action — Label/indication change, approval action, REMS modification, or withdrawal by FDA, EMA, PMDA, Health Canada, etc.
- Forecast classThe group of like forecasts this one is counted and scored with, such as FDA actions by their goal dates
- FDA action by goal date
- Scope
- Duchenne muscular dystrophy
The estimate
The number and the baselines it has to beat
The alternatives it was registered against, each with the reasoning that produced it.
ESTIMATE FROM THE SHAPE OF THE CLASS, NOT AN ENUMERATED COUNT. Approval at the goal date for resubmitted original BLAs whose first cycle ended in a complete response on effectiveness, across review types. No enumerated count exists in this repository yet.
A clock-only model: the goal date is inside the window and FDA meets most goal dates, discounted because this is a resubmission whose goal date has already been extended once.
An analyst reading the same public record on 2026-09-24 would weigh a met primary endpoint on upper limb function, CBER's acceptance of an amendment toward that indication and the unmet need in DMD against a prior complete response on effectiveness, a 3 to 9 advisory committee vote, a key secondary endpoint no longer significant, an inspection finding on trial conduct and a sponsor with no approved product. Such an analyst would call approval by the goal date less likely than not. Estimated by reconstructing that read, not by surveying analysts.
Regulatory action (FDA): offered on judgment, with no calibration figure until 15 in the class have resolved.
The clock
Dates that constrain the record
- Evidence cutoff
- 2026-09-26
Nothing after this date informed the estimate. - Registered
- 2026-09-26
Frozen and hashed. - Resolution window
- 2026-09-27 → 2026-11-22
When the adjudicator can read the outcome. - Event by
- 2026-11-22
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar
53dcbd72a686d5c74158eebc1b0aca044cd2fa9b317017ce1645fe902cdcb6ec · operator record jc-1790461082264-68c58ac7
Around this record
- This kind of deadline
- Check it, follow it
Single-claim evaluation
Waiting on the FDA for an asset of your own?
Name the claim, who decides it, and the date your team commits to: a P&T meeting, an HTA submission, a label negotiation, a deal closing. Yours is registered under the same rules as the forecast on this page.
Resolution
The rule that settles the forecast
Deramiocel is a CBER-regulated cell therapy, so openFDA's drugsfda endpoint does not list it. Resolves YES if FDA's Purple Book (https://purplebooksearch.fda.gov/) lists a licensed biological product that is deramiocel (CAP-1002) under any proprietary or proper name, with Capricor Therapeutics as licensee and an approval (license) date on or before 2026-11-22, and the CBER approval letter for that BLA names Duchenne muscular dystrophy in the indication. An indication for Duchenne muscular dystrophy as a whole, for a named part of it such as upper limb function or cardiomyopathy, or for a subpopulation of patients with it, resolves YES; so does accelerated rather than traditional approval. Resolves NO if no approval action is dated on or before 2026-11-22: a complete response letter, a further goal-date extension, withdrawal or no action. The goal date is frozen as disclosed at the cutoff; an approval dated after 2026-11-22 resolves NO as occurred_after_deadline. 2026-11-22 is a Sunday; an approval action dated on or before it counts whatever the weekday. Read no earlier than 2026-12-06. If the Purple Book has not updated by then, the CBER approval letter or FDA's list of approved cellular and gene therapy products (https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products) governs. At the cutoff that list, content current as of 2026-09-17, did not include deramiocel (SRC-004). Resolves unresolvable only if none of these can be read after the read floor. Resolves VOID, with the reason recorded, only if FDA withdraws every public record of approval actions named above without an equivalent public successor, so that no approval date for this product can be read; while any of them publishes it, the rule stands.
Source the rule names: https://purplebooksearch.fda.gov/, https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
Readers: Resolver A, clinical methodology; Resolver B, payer evidence. The same two reader roles are assigned to every forecast registered so far.
What this record will show once it resolves
Beside every result, the count for its class (FDA action by goal date): registered, resolved, unresolved, and expired. One forecast is one instance, not a rate.
- The finding
- As the rule above reads it: occurred on time, did not occur, or left unscored.
- The readers and their sources
- Two reader roles read the source named above: Resolver A, clinical methodology; Resolver B, payer evidence. Who adjudicated it is named here, with the sources they read when the adjudication lists them. Being named as a reader means applying the frozen rule to the public record; it is not support for the estimate, the method, or NextConsensus.
- The number against its baselines
- 35% beside three baselines, each scored by the same rule (squared error), so a reader can see how the record did against each of them.
Reasoning
What the number rests on
The facts it was built from are below, as they stood at the cutoff, with every source archived; each baseline above carries the reasoning that produced it. The written mechanism and the conditions that would make the proposition ill-posed were frozen with the record and are held in the ledger, which the hash on this page covers.
Frozen covariatesThe facts about the evidence and the authority's process that were frozen with the forecast at its cutoff
The state of the record at the cutoff
Recorded as they stood on 2026-09-26, including the one field whose value was unknown then. It is left unknown rather than filled in.
- review pathway
- Class 2 resubmission; the first cycle had Priority Review; goal date extended three months by a major amendment (SRC-001, SRC-003); RMAT, Orphan Drug and Rare Pediatric Disease designations (SRC-002)
- submission type
- original BLA, Class 2 resubmission after a complete response letter (allogeneic cell therapy; CBER)
- evidence class
- one placebo-controlled phase 3 trial, HOPE-3, with the phase 2 HOPE-2 trial and open-label extensions (SRC-003)
- pivotal n
- unknown — not stated in the archived sources
- endpoint type
- upper limb function on PUL 2.0, the HOPE-3 primary endpoint, met at p=0.029; key secondary LVEF at p=0.09 after a model update (SRC-003)
- approved competitors in class
- none named in the archived sources; Capricor says several therapies address certain DMD genetic mutations and that unmet need remains in established skeletal and cardiac muscle disease (SRC-003)
- months since last guideline revision
- not applicable — no guideline is the subject of this proposition
- panel cycle position
- not applicable — the act is an FDA approval action
- sponsor request status
- BLA amended with 24-month HOPE-3 open-label extension data and a refined proposed indication focused on upper limb function; CBER accepted it and moved the goal date from August 22, 2026 to November 22, 2026 (SRC-001, SRC-002)
- odac referral
- Cellular, Tissue and Gene Therapies Advisory Committee met 2026-07-29 and voted 3 for, 9 against on effectiveness for cardiomyopathy in DMD; no vote on upper limb function or overall benefit-risk (SRC-003)
- sponsor state change
- complete response letter July 2025; Form 483 with one observation on HOPE-3 trial conduct after a July 2026 BIMO inspection, response submitted (SRC-003)
Sources
Everything the estimate was built from
Each was retrieved on or before the 2026-09-26 cutoff and archived at the digest shown. If a publisher edits a page after the fact, the digest stops matching and the substitution is visible.
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SRC-001Capricor Therapeutics 8-K filed 2026-08-24 (Item 8.01). States the FDA extended the PDUFA target action date for Capricor's BLA for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22, 2026 to November 22, 2026, after classifying a submission as a major amendment that extends the review period by three months. The amendment includes 24-month open-label extension data from the Phase 3 HOPE-3 study and asks FDA to review the data in support of a refined proposed indication focused on upper limb function, the HOPE-3 primary endpoint. -
SRC-002Exhibit 99.1 to the same 8-K: Capricor press release dated 2026-08-24. States the amendment followed the July 2026 Advisory Committee meeting; that FDA's Center for Biologics Evaluation and Research (CBER) accepted the amendment for review, citing the significant unmet medical need in DMD; that the new PDUFA target action date is November 22, 2026; and that Deramiocel (CAP-1002) is allogeneic cardiosphere-derived cells holding Orphan Drug, RMAT and Rare Pediatric Disease designations in the U.S. -
SRC-003Capricor 10-Q for the quarter ended 2026-06-30, filed 2026-08-14. Records the BLA history: submitted late 2024 for the treatment of DMD, Priority Review, a Complete Response Letter in July 2025 finding the application did not meet the statutory requirement for substantial evidence of effectiveness, and a Class 2 resubmission with HOPE-3 data given a PDUFA date of August 22, 2026. Records HOPE-3 results: primary endpoint PUL 2.0 met (least squares mean difference 4.55 percentage points, p=0.029); key secondary endpoint LVEF revised to p=0.09 under an updated statistical model, so later hierarchical endpoints are nominal only. Records a July 2026 BIMO inspection ending with a Form 483 citing one observation on HOPE-3 trial conduct, answered by Capricor. Records the 2026-07-29 Cellular, Tissue and Gene Therapies Advisory Committee vote of 3 for and 9 against on whether the evidence supports effectiveness for cardiomyopathy in DMD, a narrower indication than Capricor proposed, with no vote on upper limb function or overall benefit-risk, and Capricor's plan to amend the BLA toward a refined indication focused on the HOPE-3 primary endpoint. -
SRC-004FDA page 'Approved Cellular and Gene Therapy Products' (licensed products from CBER's Office of Therapeutic Products), content current as of 2026-09-17, retrieved at the cutoff. The starting state: it does not list Deramiocel or any Capricor product.