FDA approves zeleciment rostudirsen (z-rostudirsen) for Duchenne muscular dystrophy amenable to exon 51 skipping on or before the disclosed PDUFA goal date of 2027-01-21

The proposition

What was claimed

The FDA takes an approval action on Dyne Therapeutics' Biologics License Application for zeleciment rostudirsen (z-rostudirsen, DYNE-251) for the treatment of Duchenne muscular dystrophy amenable to exon 51 skipping, with an approval action date on or before 2027-01-21, the PDUFA target action date disclosed in Dyne Therapeutics' Form 10-Q filed 2026-07-29.

Authority

US Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) (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 amenable to exon 51 skipping

The clock

Dates that constrain the record

resolution window

Today

Evidence cutoff2026-10-15 Registered2026-10-15 Window opens2026-10-16 Deadline2027-01-21

Evidence cutoff

2026-10-15

Registered

2026-10-15

Window opens

2026-10-16

Deadline

2027-01-21

Evidence cutoff

2026-10-15
Nothing after this date informed the estimate.

Registered

2026-10-15
Frozen and hashed.

Resolution window

2026-10-16 → 2027-01-21
When the adjudicator can read the outcome.

Event by

2027-01-21
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar

ledger f5b0d0e15845147745cfcc30691b9f9314ab5be9b7aa69a5bd017808ef69fbbf Copy · operator record jc-1792036249642-353eeea8

Resolution

The rule that settles the forecast

Resolves YES if the openFDA Drugs@FDA records returned by https://api.fda.gov/drug/drugsfda.json?search=openfda.generic_name:%22zeleciment%20rostudirsen%22&limit=100 and by https://api.fda.gov/drug/drugsfda.json?search=products.active_ingredients.name:%22ZELECIMENT%20ROSTUDIRSEN%22&limit=100, read together (openFDA does not always fill the generic-name field for a new application), contain an application whose ORIG submission has submission_status AP and a submission_status_date on or before 2027-01-21, and the Drugs@FDA approval letter or label for that application names Duchenne muscular dystrophy amenable to exon 51 skipping in its indication. An approval for a narrower Duchenne muscular dystrophy amenable to exon 51 skipping population than the applicant sought still resolves YES, as does accelerated rather than traditional approval. Resolves NO if no approval action is dated on or before 2027-01-21: a complete response letter, a goal-date extension, withdrawal or no action. The goal date is frozen as disclosed at the cutoff; a later extension does not move it, and an approval dated after 2027-01-21 resolves NO as occurred_after_deadline. Read no earlier than 2027-02-04. If openFDA has not refreshed by then, the Drugs@FDA web record (https://www.accessdata.fda.gov/scripts/cder/daf/, searched by the name zeleciment rostudirsen) governs. If neither lists the product, FDA's Purple Book (https://purplebooksearch.fda.gov/, searched by zeleciment rostudirsen) governs for the licensure date, with the approval letter for the indication. The application number was not public at the cutoff, so the rule reads by name; a suffix on the nonproprietary name or a proprietary name does not change the identification. Resolves unresolvable only if none of these sources 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://api.fda.gov/drug/drugsfda.json?search=openfda.generic_name:%22zeleciment%20rostudirsen%22&limit=100, https://api.fda.gov/drug/drugsfda.json?search=products.active_ingredients.name:%22ZELECIMENT%20ROSTUDIRSEN%22&limit=100, https://www.accessdata.fda.gov/scripts/cder/daf/, https://purplebooksearch.fda.gov/

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

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 2026-10-15.

review pathway

Priority Review (SRC-001, SRC-002); BLA submitted for potential U.S. Accelerated Approval based on dystrophin as a surrogate endpoint (SRC-001, SRC-004).

submission type

Original BLA (SRC-001).

evidence class

Registrational expansion cohort (REC) of the global Phase 1/2 DELIVER trial (randomized vs placebo), surrogate-endpoint basis; confirmatory Phase 3 FORZETTO initiated May 2026 (SRC-004, SRC-001).

pivotal n

DELIVER REC: 32 participants (24 z-rostudirsen 20 mg/kg Q4W, 8 placebo) (SRC-004).

endpoint type

Surrogate biomarker: change from baseline in muscle content-adjusted dystrophin at six months (met, p<0.0001; mean absolute expression 5.46% of normal) (SRC-004).

approved competitors in class

EXONDYS 51 (eteplirsen), a PMO approved for DMD amenable to exon 51 skipping (Sarepta), among four FDA-approved exon-skipping drugs (SRC-001).

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 submission planned for Q2 2026 (SRC-004); FDA acceptance with Priority Review announced July 20, 2026 (SRC-002).

odac referral

none announced in the archived sources

sponsor state change

None reported.