Will the FDA approve deramiocel in Duchenne muscular dystrophy before 30 November 2026?
deadline 2026-11-30
confidence medium
tier 4 evidence
56%PolySignal estimate
⚠ Evidence cap applied: the model proposed 45%, brought back to 56% for lack of sources solid enough to justify the departure from the base rate.
Resolution criterion
Yes if the FDA grants an approval, whatever the scope of the final indication, by 30 November 2026. A complete response letter, a further postponement, or the absence of a decision count as no.
Verified starting state
New PDUFA date of 22 November 2026, after the FDA classified the submission of 24-month open-label extension data from the HOPE-3 trial as a "major amendment". The indication has been reframed around preservation of upper-limb function. The application has already received one complete response letter and two delays. (24 August 2026)
What pushes it up
- The FDA accepted the submission of 24-month open-label extension data from the HOPE-3 trial as a major amendment, keeping the BLA active [1, 2, hand-verified starting state].
- The indication has been reframed around the preservation of upper-limb function, which may narrow and simplify the path to approval [hand-verified starting state].
What pushes it down
- The new PDUFA date of 22 November 2026 is only eight days before the 30 November 2026 deadline, leaving virtually no margin for any further delays [1, 2, hand-verified starting state].
- An FDA advisory panel and staff have previously raised questions regarding the effectiveness of deramiocel [4].
- The drug has already faced significant regulatory hurdles, including one CRL and two previous delays [hand-verified starting state].
What would move this number most
Whether the newly submitted 24-month open-label extension data is robust enough to resolve the effectiveness concerns raised by the FDA panel and staff before the tight November deadline.
How this number is built
| Deadline | 2026-11-30 |
| Base rate | 64% |
| Best evidence | tier 4 · 3 article(s) used |
| Independent runs | 56 · 56 (spread 0 pts) |
| Confidence | medium |
| Evidence cap | applied: 45% brought back to 56% |
| Last revised | 2026-09-21 22:18 |
| Model | gemini-3.5-flash |
Sources consulted
Probability history
2026-09-21 22:18 56% 8 article(s) on file · gemini-3.5-flash
Reasons not recorded for this revision (detailed history has only been kept since recently).
2026-09-21 21:36 56% 8 article(s) on file · openai/gpt-oss-120b (Groq)
Reasons not recorded for this revision (detailed history has only been kept since recently).
2026-09-19 07:33 77% 7 article(s) on file · gemini-3-flash-preview
Reasons not recorded for this revision (detailed history has only been kept since recently).