Registered forecast
78% registered probability event by 2027-02-28 Unresolved
FDA grants traditional (full) approval of VOXZOGO (vosoritide) for achondroplasia on or before the disclosed PDUFA goal date of 2027-02-28
FF-062 Registered - Registered probability
- 78%
- Decision by
- 2027-02-28
- Reading window
- 2026-10-22 → 2027-02-28
- Decision stage
- 5 Guideline
- 6 Regulatory
- 7 Coverage
- Baselines it has to beat
-
- Authority base rate 85%
- Calendar cycle 80%
- Analyst proxy 82%
nextconsensus.com/forecast/ff-062/ registry snapshot 2026-11-07 Share image
NextConsensus registered this probability on 2026-11-04. It is one of 35 registered FDA goal-date forecasts.
The proposition
What was registered
The FDA takes an approval action on a supplement to NDA 214938 (vosoritide, VOXZOGO; applicant BioMarin Pharmaceutical) that converts VOXZOGO’s achondroplasia indication from accelerated approval to traditional approval, with an approval action date on or before 2027-02-28, the PDUFA target action date disclosed in Exhibit 99.1 to BioMarin’s Form 8-K filed 2026-08-06.
- Who decides
- US Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) (US)
- Decision stageThe kind of body that has to act: a guideline panel, a regulator, or a payer. 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.
- Decision typeThe type of decision this forecast is counted with, such as FDA actions by their goal dates
- FDA action by goal date
- Scope
- Achondroplasia in children (traditional approval)
The estimate
The number and the baselines it has to beat
The estimate that the named body takes the action by the deadline, from what was public at the cutoff.
The alternatives it was registered against, each with the reasoning that produced it. They were set at registration: how often this kind of decision happens on time, what the calendar alone suggests, and a reading of the same public record without this method. Each one's reasoning shows whether it is a count of past decisions or an estimate.
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; no count of accelerated-to-traditional conversions decided at a disclosed goal date was made. The calendar’s own met-goal-date share for disclosed PDUFA dates does not exist yet (docs/foundations/short-horizon-reference-classes.md §4.1).
A clock-only model: the goal date is inside the window, FDA meets most goal dates, and most first-cycle supplement actions are approvals. With no evidence input it cannot tell a strong application from a weak one.
An analyst reading the same filings on 2026-10-21 would weigh five years on the market under accelerated approval, an all-ages label, FDA’s acceptance of the sNDA and a large commercial base, against confirmatory evidence that is open-label extension data compared with natural history rather than a randomized trial. 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 method and every record are public, so you can check the work.
The clock
Dates that constrain the record
- Evidence as of
- 2026-10-21
Nothing after this date informed the estimate. - Registered
- 2026-11-04
Frozen and hashed. - Reading window
- 2026-10-22 → 2027-02-28
When the outcome can be read against the rule. - Event by
- 2027-02-28
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar
be93515bf0714019ff3b0a1573777d7f27addf881cb672a151f79200c85c5548 · operator record jc-1793799400128-8ae141ef Around this record
- This kind of deadline
- Check it, follow it
Confidential Asset & Milestone Evaluation
Evaluating an asset or milestone that the FDA could move?
Name the asset, the deciding authority, and the date your team commits to: a P&T review, an HTA submission, a label negotiation, a deal closing, or a milestone dispute. Your stake need not be direct sponsorship — an asset you evaluate, finance, advise on, or rely upon is enough. Every commercial evaluation applies the same rigorous pre-registration and primary-source verification shown on this page, delivered privately under mutual NDA.
Resolution
The rule that settles the forecast
Resolves YES if the openFDA Drugs@FDA record for application NDA214938 (https://api.fda.gov/drug/drugsfda.json?search=application_number:%22NDA214938%22) contains a submission with submission_type SUPPL, submission_status AP and a submission_status_date on or before 2027-02-28, and either the Drugs@FDA approval letter for that supplement states that it provides for traditional (full) approval of the achondroplasia indication, or the label approved with it states the achondroplasia indication without the accelerated-approval statement the label carried at the cutoff (that the indication is approved under accelerated approval based on an improvement in annualized growth velocity, and that continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials; SRC-003). The supplement is identified by that content, not by its number or class code. A conversion that covers part of the achondroplasia age range still resolves YES. A supplement that adds hypochondroplasia, or any other indication, does not count unless the same letter or label also removes the accelerated-approval statement from the achondroplasia indication: BioMarin submitted its hypochondroplasia sNDA separately in July 2026 (SRC-002). At the cutoff the latest approved supplement was SUPPL 4, a labeling supplement approved 2024-11-04 (SRC-004). Resolves NO if no qualifying approval action is dated on or before 2027-02-28: 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 a qualifying approval dated after 2027-02-28 resolves NO as occurred_after_deadline. 2027-02-28 is a Sunday; an approval action dated that day still counts. Read no earlier than 2027-03-14; if openFDA has not refreshed, the Drugs@FDA web record for NDA214938 (https://www.accessdata.fda.gov/scripts/cder/daf/) governs. Resolves unresolvable only if neither source 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=application_number:%22NDA214938%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
- 78% 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 2026-10-21, including the 3 fields whose values were unknown then. Those are left unknown rather than filled in.
- review pathway
- unknown — the archived sources do not name the review designation; the sNDA was accepted in July 2026 with a goal date of 2027-02-28 (SRC-001, SRC-002). Center CDER (NDA listed in openFDA drugsfda, SRC-004).
- submission type
- Supplemental NDA to NDA 214938 for full (traditional) approval of the existing achondroplasia indication, converting it from accelerated approval (SRC-001, SRC-002, SRC-003).
- evidence class
- Open-label extension studies of VOXZOGO compared with available natural history, as BioMarin described its plan for the confirmatory requirement (SRC-002, SRC-003); the archived sources do not describe the submitted dataset further.
- pivotal n
- unknown — not stated in the archived sources
- endpoint type
- Accelerated approval rested on annualized growth velocity, a surrogate (SRC-003); the clinical-benefit measure the conversion relies on is not named in the archived sources.
- approved competitors in class
- unknown — the archived sources name no other approved achondroplasia therapy
- 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 for full approval accepted, announced July 2026, PDUFA target action date 2027-02-28 (SRC-001, SRC-002). A separate hypochondroplasia sNDA was submitted in July 2026 (SRC-002). The latest approved supplement on the application at the cutoff was SUPPL 4, labeling, 2024-11-04 (SRC-004).
- odac referral
- none announced in the archived sources
- sponsor state change
- BioMarin acquired Amicus’s commercial products on 2026-04-27 (SRC-001, SRC-002) and discontinued VOXZOGO development in Noonan syndrome on 2026-09-15 for reasons it says were not related to safety or efficacy (SRC-005).
Sources
Everything the estimate was built from
Each was retrieved on or before the 2026-10-21 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-001BioMarin Form 8-K Exhibit 99.1, filed 2026-08-06: Q2 2026 results release. In July FDA accepted BioMarin's sNDA for full approval of VOXZOGO in children with achondroplasia, PDUFA target action date February 28, 2027; a separate hypochondroplasia sNDA was submitted after CANOPY-HCH-3 met its primary endpoint; full-year 2026 VOXZOGO revenue guidance raised to at least 1 billion dollars; Amicus products being integrated. -
SRC-002BioMarin Form 10-Q for the quarter ended 2026-06-30, filed 2026-08-06: FDA accepted the sNDA for full approval of VOXZOGO in achondroplasia, PDUFA 2027-02-28; the hypochondroplasia sNDA was submitted in July 2026; VOXZOGO is approved in the U.S. under accelerated approval, and BioMarin intends to meet the confirmatory requirement with its open-label extension studies compared to available natural history; Amicus commercial products acquired 2026-04-27. -
SRC-003BioMarin Form 10-K for 2025, filed 2026-02-26: VOXZOGO is approved in the U.S. for achondroplasia in children with open growth plates of all ages, under accelerated approval based on an improvement in annualized growth velocity; continued approval may be contingent upon verification and description of clinical benefit in confirmatory studies, which BioMarin intends to meet with open-label extension studies compared to available natural history. -
SRC-004openFDA drugsfda application_number:"NDA214938", retrieved 2026-10-21 (meta last_updated 2026-10-20): VOXZOGO, applicant BioMarin; ORIG-1 (new molecular entity) approved 2021-11-19, SUPPL 2 efficacy approved 2023-10-20, SUPPL 4 labeling approved 2024-11-04. No approval after 2024-11-04. -
SRC-005BioMarin Form 8-K (Item 8.01), filed 2026-09-15: BioMarin discontinued development of VOXZOGO for children with Noonan syndrome after assessing study feasibility and the treatment landscape; it states the decision was not related to the safety or efficacy of VOXZOGO. No change to the achondroplasia application.