Registered forecast
75% registered probability event by 2026-11-25 Unresolved
FDA approves venglustat for type 3 Gaucher disease on or before the disclosed PDUFA goal date of 2026-11-25
FF-027 Registered - Registered probability
- 75%
- Event by
- 2026-11-25
- Resolution window
- 2026-10-07 → 2026-11-25
- Target rung
- 5 Guideline
- 6 Regulatory
- 7 Coverage
- Baselines it has to beat
-
- Authority base rate 85%
- Calendar cycle 80%
- Analyst proxy 75%
nextconsensus.com/forecast/ff-027/ registry snapshot 2026-10-06 Share image
NextConsensus registered this probability on 2026-10-06. It is one of 14 registered FDA goal-date forecasts.
The proposition
What was claimed
The FDA takes an approval action on Sanofi's New Drug Application for venglustat for the treatment of type 3 Gaucher disease, with an approval action date on or before 2026-11-25, the target action date disclosed in Sanofi's 2026 half-year management report, furnished on Form 6-K on 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
- Type 3 Gaucher 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. Applications built on positive randomized controlled trials are approved at their first goal date in a large majority of cases. The calendar's own met-goal-date share for disclosed PDUFA dates does not exist yet.
A clock-only model: the goal date is inside the window, FDA meets most goal dates, and most first-cycle actions on applications like this are approvals. With no evidence input it cannot tell a strong application from a weak one.
A rare-disease analyst reading the same filings on 2026-10-01 would weigh priority review, Breakthrough Therapy designation and a phase 3 that met both neurological co-primary endpoints against an active comparator, against a small trial and failures of the same drug in Fabry disease. 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
- 2026-10-06
Nothing after this date informed the estimate. - Registered
- 2026-10-06
Frozen and hashed. - Resolution window
- 2026-10-07 → 2026-11-25
When the adjudicator can read the outcome. - Event by
- 2026-11-25
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar
e47801540786a6fe2ed9a4a168e3e7923e30b1e5efe6a34cee6a64d3108081f8 · operator record jc-1791309828469-68a8e289
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 records returned by https://api.fda.gov/drug/drugsfda.json?search=openfda.generic_name:%22venglustat%22&limit=100 and by https://api.fda.gov/drug/drugsfda.json?search=products.active_ingredients.name:%22VENGLUSTAT%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 2026-11-25, and the Drugs@FDA approval letter or label for that application names type 3 Gaucher disease in its indication, with Sanofi or a Sanofi subsidiary as applicant. An approval for a narrower type 3 Gaucher disease population, or for its neurological manifestations only, still resolves YES. Resolves NO if no approval action is dated on or before 2026-11-25: 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 2026-11-25 resolves NO as occurred_after_deadline. Read no earlier than 2026-12-09. If openFDA has not refreshed by then, the Drugs@FDA web record (https://www.accessdata.fda.gov/scripts/cder/daf/, searched by the name venglustat) governs. 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:%22venglustat%22&limit=100, https://api.fda.gov/drug/drugsfda.json?search=products.active_ingredients.name:%22VENGLUSTAT%22&limit=100, 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
- 75% 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 2026-10-06.
- review pathway
- priority review (SRC-001, SRC-002, SRC-003); Breakthrough Therapy and Fast Track designations and US orphan designation for GD3 (SRC-002)
- submission type
- original NDA for a new investigational molecule (SRC-002)
- evidence class
- one randomized, double-blind, double-dummy, active-comparator (ERT) Phase 3 study, LEAP2MONO (SRC-002)
- pivotal n
- 43 patients randomized 1:1 (SRC-002)
- endpoint type
- co-primary: change in SARA modified total score and in RBANS total scale index score vs ERT from baseline to week 52; both met, plus 3 of 4 key secondary endpoints (SRC-002)
- approved competitors in class
- Sanofi states there are currently no approved targeted treatments that specifically address the neurological symptoms of GD3 (SRC-002); Sanofi's Cerezyme and Cerdelga form its existing Gaucher disease franchise (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
- priority review granted May 2026 (SRC-001); NDA submission date not stated
- odac referral
- none announced in the archived sources
- sponsor state change
- none recorded
Sources
Everything the estimate was built from
Each was retrieved on or before the 2026-10-06 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-001Sanofi Form 6-K (furnished 2026-07-30), exhibit: 2026 half-year management report (latest Sanofi filing found that states the date). States that in May FDA granted priority review to the NDA for venglustat for type 3 Gaucher disease and that 'The target action date for the FDA decision is November 25, 2026'; also states venglustat did not meet the primary endpoint in the PERIDOT and CARAT Phase 3 studies in Fabry disease. -
SRC-002Sanofi Form 6-K Exhibit 99.1, furnished 2026-06-08: press release dated May 28, 2026 'Sanofi's venglustat accepted for priority review in the US to treat type 3 Gaucher disease'. States the target action date (November 25, 2026), describes the LEAP2MONO Phase 3 study (43 patients randomized 1:1 vs ERT; both primary endpoints and three of four key secondary endpoints met) and prior Breakthrough Therapy, Fast Track and orphan designations. -
SRC-003Sanofi Form 6-K (furnished 2026-07-30), Q2 2026 results press release. Lists 'venglustat – GD3 – priority review (US)' and states the target action date for the FDA decision is November 25, 2026. -
SRC-004openFDA drugsfda API response for generic name 'venglustat', retrieved 2026-10-01: 404 NOT_FOUND 'No matches found!'.