Registered forecast
85% registered probability event by 2027-01-30 Unresolved
FDA approves the leniolisib (JOENJA) pediatric supplement for APDS in children weighing 13 kg to 27 kg on or before the disclosed PDUFA goal date of 2027-01-30
FF-046 Registered - Registered probability
- 85%
- Event by
- 2027-01-30
- Resolution window
- 2026-10-16 → 2027-01-30
- Target rung
- 5 Guideline
- 6 Regulatory
- 7 Coverage
- Baselines it has to beat
-
- Authority base rate 85%
- Calendar cycle 80%
- Analyst proxy 85%
nextconsensus.com/forecast/ff-046/ registry snapshot 2026-10-15 Share image
NextConsensus registered this probability on 2026-10-15. It is one of 26 registered FDA goal-date forecasts.
The proposition
What was claimed
The FDA takes an approval action on a supplement to NDA 217759 (leniolisib, JOENJA; applicant Pharming) whose approved labeling provides dosing for patients with activated phosphoinositide 3-kinase delta syndrome (APDS) weighing 13 kg to less than 27 kg, with an approval action date on or before 2027-01-30, the PDUFA target action date disclosed in Pharming's Form 6-K furnished 2026-09-25.
- 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
- APDS in children 4 years and older weighing 13 kg to less than 27 kg
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 filings on 2026-10-01 would weigh priority review and FDA's approval of the previous pediatric supplement on 2026-09-11 against an open-label single-arm basis and a chief executive's sudden departure. 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
- 2026-10-15
Nothing after this date informed the estimate. - Registered
- 2026-10-15
Frozen and hashed. - Resolution window
- 2026-10-16 → 2027-01-30
When the adjudicator can read the outcome. - Event by
- 2027-01-30
The final qualifying action date; the frozen resolution rule defines what counts. Add to calendar
7d58d3ccc4f675de696cdbed410ac76e0198710c03c6c10f60f7f3691c5f4822 · operator record jc-1792036524728-eb86fb81
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 NDA217759 (https://api.fda.gov/drug/drugsfda.json?search=application_number:%22NDA217759%22) contains a submission with submission_type SUPPL, submission_status AP and a submission_status_date on or before 2027-01-30, and the Drugs@FDA approval letter or label for that supplement provides dosing for patients with APDS weighing 13 kg to less than 27 kg. The supplement is identified by that content, not by its number or class code. At the cutoff the latest approved supplement was SUPPL 6, approved 2026-09-11, which added dosing for children aged 4 to 11 weighing 27 kg or more and is not this supplement (SRC-003). An approval covering part of the 13 kg to 27 kg range, or a narrower age range, still resolves YES. Resolves NO if no approval action is dated on or before 2027-01-30: 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 2027-01-30 resolves NO as occurred_after_deadline. 2027-01-30 is a Saturday; an approval action dated that day still counts. Read no earlier than 2027-02-13; if openFDA has not refreshed, the Drugs@FDA web record for NDA217759 (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:%22NDA217759%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
- 85% 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 2026-10-15, including the one field whose value was unknown then. It is left unknown rather than filled in.
- review pathway
- Priority Review (SRC-001). Center CDER is the drafter's understanding (NDA listed in openFDA drugsfda), unverified.
- submission type
- Supplemental NDA (efficacy; new pediatric population/lower doses) (SRC-001, SRC-002).
- evidence class
- Open-label, multinational, single-arm Phase III study in children aged 4–11 years plus clinical pharmacology assessments supporting lower-weight dosing (SRC-001).
- pivotal n
- unknown — not stated in the archived sources
- endpoint type
- Pharmacodynamic/clinical markers over 12 weeks: reduced lymphadenopathy and increased naive B cells (SRC-001).
- approved competitors in class
- None stated: Joenja is described as "the first and only targeted treatment" of APDS (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
- sNDA submitted July 30, 2026 (SRC-002); acceptance and Priority Review announced September 25, 2026 (SRC-001). A prior sNDA (40 mg and 50 mg doses, ages 4–11 weighing ≥27 kg) was approved September 11, 2026 (SRC-002, SRC-003).
- odac referral
- none announced in the archived sources
- sponsor state change
- CEO Fabrice Chouraqui stepped down with immediate effect on September 29, 2026; interim co-CEOs appointed (SRC-004).
Sources
Everything the estimate was built from
Each was retrieved on or before the 2026-10-15 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-001Pharming Form 6-K with press release dated 2026-09-25. Announces FDA acceptance and Priority Review of the sNDA for lower doses of Joenja for children aged 4 years and older weighing between 13 kg and 27 kg with APDS; PDUFA target action date January 30, 2027; supporting single-arm Phase III data. -
SRC-002Pharming Form 6-K with press release dated 2026-09-11. Announces FDA approval of the sNDA for 40 mg and 50 mg doses for children 4–11 years weighing at least 27 kg, and that on July 30 Pharming submitted a separate sNDA for lower doses for patients aged 4 to 11 weighing 13 kg to less than 27 kg. -
SRC-003openFDA drugsfda application_number:"NDA217759", retrieved 2026-10-01 (meta last_updated 2026-09-29): ORIG-1 (2023-03-24), SUPPL-5 labeling (2025-05-28), SUPPL-6 efficacy approved 2026-09-11. -
SRC-004Pharming Form 6-K with press release dated 2026-09-29 (latest before cutoff). Announces CEO Fabrice Chouraqui stepping down with immediate effect and appointment of interim co-CEOs.