Registered forecast
72% registered probability event by 2026-11-01 Unresolved
FDA approves the mitapivat sickle cell disease supplement to NDA 216196 on or before the disclosed PDUFA goal date of 2026-11-01
FF-012 Registered - Registered probability
- 72%
- Event by
- 2026-11-01
- Resolution window
- 2026-09-27 → 2026-11-01
- Target rung
- 5 Guideline
- 6 Regulatory
- 7 Coverage
- Baselines it has to beat
-
- Authority base rate 85%
- Calendar cycle 80%
- Analyst proxy 70%
nextconsensus.com/forecast/ff-012/ 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 a supplement to NDA 216196 (mitapivat, PYRUKYND; applicant Agios) whose approved labeling includes sickle cell disease, with an approval action date on or before 2026-11-01, the PDUFA goal date disclosed in Agios' Form 10-Q filed 2026-07-30.
- 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
- Sickle cell disease
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. Efficacy supplements to approved drugs are approved at their first goal date more often than original applications. The calendar's own count does not exist yet.
A clock-only model: the goal date is inside the window, FDA meets most goal dates, and most first-cycle supplement actions are approvals.
An analyst reading the same public record on 2026-09-22 would weigh an established drug and a confirmatory trial already running against the uncertainty of accelerated approval in sickle cell disease on a surrogate endpoint. The analyst would call approval by the date more likely than not. Estimated by reconstructing that read.
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 → 2026-11-01
When the adjudicator can read the outcome. - Event by
- 2026-11-01
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar
d9729672f768800f59c0828a962a9f8b7911e1225296817037a10b5de6b5049d · operator record jc-1790461077743-76b89c3f
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
Resolves YES if the openFDA Drugs@FDA record for application NDA216196 (https://api.fda.gov/drug/drugsfda.json?search=application_number:%22NDA216196%22) contains a submission with submission_type SUPPL, submission_status AP and a submission_status_date on or before 2026-11-01, and the Drugs@FDA label or letter for that supplement includes sickle cell disease in the indication. Approval under accelerated or traditional approval both count, as does a narrower sickle cell population. Resolves NO if no such supplement is approved on or before 2026-11-01: a complete response letter, a goal-date extension, withdrawal or no action. An approval dated after 2026-11-01 resolves NO as occurred_after_deadline. If the sickle cell indication is approved under a new NDA rather than a supplement, it still counts, read by the same fields. Read no earlier than 2026-11-15; if openFDA has not refreshed, the Drugs@FDA web record for NDA 216196 governs. At the cutoff the latest approved supplement was SUPPL 6, dated 2026-04-08 (SRC-002). 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=application_number:%22NDA216196%22, https://www.accessdata.fda.gov/scripts/cder/daf/
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
- 72% 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 3 fields whose values were unknown then. Those are left unknown rather than filled in.
- review pathway
- Priority Review; accelerated approval pathway (SRC-001)
- submission type
- efficacy supplement to NDA 216196
- evidence class
- completed double-blind phase 3 study, RISE UP (SRC-001); confirmatory phase 3 REIGNITE initiated
- pivotal n
- unknown — not stated in the archived sources
- endpoint type
- unknown in the archive — accelerated approval implies a surrogate endpoint; REIGNITE measures transfusion burden (SRC-001)
- approved competitors in class
- unknown in the archive
- 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
- sNDA submitted May 2026, accepted with Priority Review (SRC-001)
- odac referral
- none announced in the archived sources
- sponsor state change
- latest approved supplement to NDA 216196 is SUPPL 6, 2026-04-08 (SRC-002)
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.
-
SRC-001Agios 10-Q for the quarter ended 2026-06-30, filed 2026-07-30. States Agios submitted an sNDA for U.S. accelerated approval of mitapivat in sickle cell disease in May 2026, accepted with priority review and a PDUFA goal date of November 1, 2026. -
SRC-002openFDA Drugs@FDA record for NDA216196 (PYRUKYND, mitapivat) retrieved at the cutoff. The starting state: the latest approved supplement is SUPPL 6, AP 2026-04-08; no sickle cell supplement is approved.