Oncofusion

start / graph / reg-rare-pediatric-disease-prv

Explainerstrong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.Q8-impact

The rare paediatric disease priority review voucher: a $150-200m bearer bond, lapsed for 14 months, now live to 2029

also called RPD PRV · rare pediatric disease priority review voucher · Creating Hope Act voucher · PRV

A company that gets a drug approved for a rare children's disease receives a voucher that makes any one future FDA review faster, and it can sell that voucher to anyone - recent ones went for $150-200 million. The programme stopped issuing new designations in December 2024, and Congress restarted it on 3 February 2026 with a new cut-off of 30 September 2029.

The same thing, for a scientist

The rare paediatric disease priority review voucher programme sits at FDCA section 529 (21 U.S.C. 360ff), created by FDASIA 2012. A sponsor first obtains rare paediatric disease designation - for a serious or life-threatening condition whose serious manifestations primarily affect individuals from birth to 18 years and that is also a rare disease - and, on approval of an application for that product, receives a fully transferable voucher entitling the bearer to priority (six-month) review of one future application. Redeeming it costs a separate user fee, $1,962,472 in FY2026, on top of the ordinary PDUFA fee. The secondary market is the real incentive: reported transactions run from $21.2m to $350m, with 2025-26 deals clustering at $150-200m. FDA's authority to award new vouchers lapsed on 2024-12-20 and was restored by section 6604 of the Consolidated Appropriations Act, 2026 (signed 2026-02-03), which carries the Mikaela Naylon Give Kids a Chance Act; FDA may not award vouchers under this programme after 2029-09-30, and the previously feared 2026-09-30 approval deadline is moot. No public record was found of a rare paediatric disease designation or a voucher for revumenib or ziftomenib.

Go deeper
What the programme requires

Two steps, in order 2:

1. Rare paediatric disease designation. The sponsor asks FDA to confirm that the product treats or prevents a rare paediatric disease, defined in FDCA 529(a)(3) as a disease that is (i) serious or life-threatening, with serious or life-threatening manifestations that primarily affect individuals aged from birth to 18 years, and (ii) a rare disease or condition within the meaning of the Orphan Drug Act (fewer than 200,000 US persons, or the cost-recovery test).

2. Award on approval. If FDA then approves a marketing application for that product for that rare paediatric disease, and the application meets the statutory conditions, FDA awards a priority review voucher.

The voucher entitles its bearer to priority review - roughly six months from filing rather than ten - of one subsequent human drug application, and it is fully transferable without limit on the number of transfers. It is, functionally, a bearer instrument.

One practical point that shapes what can be verified: rare paediatric disease designations are not publicly disclosable. FDA's own Office of Orphan Products Development says so explicitly in the only published analysis of the designation arm of the programme 4. Voucher awards are public; designations are not. This asymmetry matters below.

What it is worth

The redemption fee. Using a voucher is not free. For FY2026 (effective 2025-10-01 through 2026-09-30) the priority review user fee for a tropical disease, rare paediatric disease or material-threat MCM voucher is $1,962,472, payable in addition to any PDUFA fee due on the application 3. FDA published an FY2027 rate on 2026-09-10; that figure was not verified for this node and will govern from 2026-10-01.

The resale value. This is where the incentive actually lives. Reported transaction values 67:

YearSellerReported price
2015United Therapeutics to AbbVie$350m (highest ever reported)
2023seller to Novartis$21.2m (lowest ever reported)
2025Abeona Therapeutics (Zevaskyn)$155m (sale closed) 7
2025Bavarian Nordic (chikungunya vaccine)$160m
2026Jazz Pharmaceuticals (Modeyso)$200m, announced at J.P. Morgan

PTC Therapeutics, Zevra and Acadia each reported $150m, and Ipsen $158m, in the same period 6. The clustering at $150m+ is widely attributed to supply anxiety after the December 2024 lapse - a scarcity premium created by Congress's own inaction, which is an odd way to price a child-health incentive.

Scale of the programme. Reporting around the 2026 reauthorisation puts the historical total at 63 rare paediatric disease vouchers awarded across 47 diseases 6. FDA's own analysis of the designation arm counted 569 rare paediatric disease designations between 2013 and 2022, of which 18% (n=105) were oncology, behind neurology (26%) and metabolism (23%) 4.

CURRENT STATUS as of 2026-09-18 - the part that goes stale fastest

The programme is live. Here is the sequence, dated:

  • 2024-12-20 - FDA's authority to grant new rare paediatric disease designations lapsed. Vouchers could still be awarded for products designated before that date if approved by 2026-09-30.
  • 2025 through early 2026 - repeated failed reauthorisation attempts, centred on the Give Kids a Chance Act (H.R. 1262 in the 119th Congress, the Mikaela Naylon Give Kids a Chance Act, combining the original Give Kids a Chance Act and the Creating Hope Reauthorization Act) 5.
  • 2026-02-03 - the Consolidated Appropriations Act, 2026 was signed into law. Its section 6604 carries the Give Kids a Chance Act provisions and reauthorises the programme. Its section 6605 separately fixed orphan drug exclusivity scope (see reg-orphan-drug-designation) 5.
  • Today, 2026-09-18 - FDA's own programme page states that "FDA may not award any PRVs under this program after September 30, 2029", and describes the sunset as established by the Consolidated Appropriations Act, 2026; the page is marked content current as of 2026-04-29 and gives active instructions for requesting designations 1. FDA is granting new rare paediatric disease designations again. The 2026-09-30 approval deadline that governed during the lapse is superseded and is no longer the operative date.

One nuance worth keeping: commentary on the new law notes that the statute does not impose a separate deadline for obtaining designation, only the 2029-09-30 deadline on awarding vouchers 5. A sponsor designated in 2028 whose approval lands in 2030 gets nothing.

The Commissioner's National Priority Voucher (CNPV) pilot is real, and is a different animal. FDA runs it under a page marked content current as of 2026-08-26 9. Announced June 2025, it targets a 1-2 month review against five declared national priorities (public-health crisis response, innovative breakthrough therapies, large unmet medical need, onshoring and supply-chain resilience, affordability), with enhanced communications, rolling review and tumour-board-style meetings. Crucially, CNPV vouchers are nontransferable - they cannot be sold 9. They are a discretionary agency instrument, not a statutory tradable asset, and they are not a substitute for an RPD voucher for a small sponsor whose whole financing plan depends on selling one.

Has it been used for a KMT2A indication? No public evidence

Revumenib (Revuforj, approved 2024-11-15 for R/R KMT2A-translocated acute leukaemia, patients 1 year and older). FDA's approval announcement records priority review and fast track designation and does not mention a rare paediatric disease designation or a priority review voucher 1011. Syndax's own release on the approval likewise makes no voucher claim. No voucher was awarded.

Ziftomenib (Komzifti, approved 2025-11-13). Its approved indication is adults with R/R NPM1-mutant AML - by construction it cannot satisfy the "primarily affect individuals from birth to 18" test. No voucher.

Two caveats on how strong this negative is:

  1. Designations are not disclosable 4, so "no rare paediatric disease designation was found" is weak evidence that none was granted. Voucher awards are public, so "no voucher" is strong.
  2. Whether R/R KMT2A-translocated acute leukaemia would even qualify is genuinely arguable. KMT2A rearrangement defines the great majority of infant ALL, but the approved population is not primarily paediatric: in the pivotal AUGMENT-101 KMT2A cohort the median age was 37 years (range 1-79) with only 24% under 17 12. "Primarily affects individuals from birth to 18" is a statutory test about the disease, not the trial - but a sponsor building a designation request would be arguing uphill against its own registrational demographics.

So: the single most paediatric fusion-driven leukaemia in oncology produced the first approved menin inhibitor and generated no rare paediatric disease voucher. That is the finding.

Facts and figures
programmerare pediatric disease priority review voucher
jurisdictionUS
statuteFDCA 529 (21 U.S.C. 360ff), created by FDASIA 2012
voucher transferabletrue
priority review months6
standard review months10
fy2026 prv user fee usd1962472
reported sale price min usd21200000
reported sale price max usd350000000
vouchers awarded historical63
rpd designations 2013 2022569
rpd designations oncology PCT18
designation authority lapsed2024-12-20
reauthorised2026-02-03
reauthorising lawConsolidated Appropriations Act, 2026, section 6604 (Mikaela Naylon Give Kids a Chance Act)
current sunset award deadline2029-09-30
superseded sunset2026-09-30
programme live on 2026 09 18true
cnpv pilot realtrue
cnpv transferablefalse
used for kmt2afalse
revumenib prv awardedfalse
ziftomenib prv awardedfalse
as of2026-09-18
recent sale prices usd
ptc150,000,000ptc: 150000000zevra150,000,000zevra: 150000000acadia150,000,000acadia: 150000000abeona_2025155,000,000abeona_2025: 155000000ipsen158,000,000ipsen: 158000000bavarian_nordic_2025160,000,000bavarian_nordic_2025: 160000000jazz_2026200,000,000jazz_2026: 200000000
recent sale prices usd — the numbers behind the chart
{
  "abeona_2025": 155000000,
  "bavarian_nordic_2025": 160000000,
  "jazz_2026": 200000000,
  "ptc": 150000000,
  "zevra": 150000000,
  "acadia": 150000000,
  "ipsen": 158000000
}
How this could bite us back
  • The voucher rewards an approval, not a child treated. Nothing in FDCA 529 requires that the paediatric formulation be commercially available at launch, that any minimum number of children be dosed, or that the paediatric indication ever be commercially supported. Revuforj itself illustrates the gap from the other direction: FDA's own approval announcement notes that for patients weighing under 40 kg the drug was initially available only through an expanded access programme because commercial supply was delayed. The smallest patients waited after approval, not before it.
  • A sponsor can win a rare paediatric disease voucher for a product whose actual market is adults, then sell the voucher to accelerate a completely unrelated blockbuster. The $150-200m goes to the company; the accelerated review goes to whatever drug the buyer chooses; neither has to have anything to do with children. The instrument is fungible by design, and fungibility is exactly what severs the reward from the benefit.
  • Goh, Outterson and Kesselheim looked at the tropical-disease arm of the same instrument and found FDA approval arrived a median of 8.7 years after the drug was already approved or in use in an endemic country - the voucher paid for paperwork on old medicines, not for new science. The mechanism is identical in the paediatric arm: the trigger is an approval event, and approval events can be manufactured from existing evidence.
  • Congress let the programme lapse for 14 months, from 2024-12-20 to 2026-02-03. Paediatric oncology programmes run on 8-to-12-year timescales. An incentive whose existence depends on an appropriations rider is not something a small sponsor can underwrite an infant leukaemia programme against - and the market noticed, bidding voucher prices to record levels precisely because supply became uncertain. Policy volatility priced the asset up and the incentive down at the same time.
  • The new sunset, 2029-09-30, is three years away. No programme that begins today in infant KMT2A-rearranged leukaemia can reach approval before it. For the population this node is about, the reauthorised voucher is already out of reach unless Congress extends it again - which is to say the incentive works best for sponsors who were already nearly finished, and not at all for the ones who have not started.
  • The most paediatric fusion-driven leukaemia in oncology - KMT2A rearrangement, which defines the majority of infant ALL - produced the first approved menin inhibitor and generated no rare paediatric disease voucher at all. Meanwhile the adult NPM1 slice of the same drug class produced two approvals in thirteen months. If the voucher is supposed to be pulling development toward children, it did not pull here.
Still open
  1. Did Syndax ever request or obtain rare paediatric disease designation for revumenib? Designations are not publicly disclosable, so this cannot be answered from public sources - it can only be answered by asking the sponsor.
  2. What is the FY2027 priority review voucher user fee, effective 2026-10-01? The Federal Register notice exists (doc. 2026-18464, published 2026-09-10) but was not readable here.
  3. Would FDA accept that relapsed or refractory KMT2A-translocated acute leukaemia 'primarily affects individuals from birth to 18 years' given that the registrational cohort had a median age of 37? There appears to be no published FDA precedent on a fusion-defined leukaemia subtype spanning infancy to old age.
  4. Does a 2029-09-30 award deadline leave enough runway to incentivise any programme that starts now? A first-in-class paediatric leukaemia drug takes longer than three years from IND to approval.
Every source behind this entry (12)
  1. E1established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database

    As of 2026-09-18 FDA's rare paediatric disease designation and priority review voucher programme page states that FDA may not award any PRVs under the programme after September 30, 2029, attributes that sunset to the Consolidated Appropriations Act, 2026, gives active instructions for requesting designations, and is marked content current as of 2026-04-29.

    What this evidence cannot show

    Retrieved 2026-09-18. The page did not restate the 2024-12-20 lapse or define 'rare pediatric disease' in the retrieved text; those come from E2 and E5. A page marked current as of 2026-04-29 is nearly five months old on the date of writing.

    Evidence that argues against this (1)
    • A fee, an exclusivity date or a programme status is a dated fact with a short shelf life: fee schedules are reset every fiscal year, exclusivity dates move on paediatric extension or litigation, and programme sunsets have been extended by Congress before. Anything planned on these has to re-query them. The PRV sunset in particular has been extended by Congress repeatedly since the programme began, so a stated end date is a legislative default rather than a forecast. U.S. Food and Drug Administration 2026
  2. E2established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database

    FDCA section 529 (21 U.S.C. 360ff) defines a rare paediatric disease as a serious or life-threatening disease whose serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and which is also a rare disease or condition, and provides for award of a transferable priority review voucher on approval of a rare paediatric disease product application.

    21 U.S.C. 360ff - Priority review to encourage treatments for rare pediatric diseases · United States Code · Office of the Law Revision Counsel, US House of Representatives 2026
    What this evidence cannot show

    Statutory text; the prelim US Code may lag the February 2026 amendments (see E5). The definition itself was not changed by the reauthorisation.

    Evidence that argues against this (1)
    • A statute or a programme page states what the law PERMITS, not how the agency exercises the discretion the law gives it. Every operative term here — serious condition, substantial improvement, unmet need, plausible medical use — is applied case by case and the reasoning is not published, so the text bounds the outer edge of what could happen and predicts nothing about a particular application. The definition turns on manifestations PRIMARILY affecting people aged birth to 18, which for KMT2A-rearranged leukaemia across all ages is precisely the contested question: the molecular lesion occurs at every age and the infant form is the one this graph is about. U.S. Food and Drug Administration 2026
  3. E3established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database

    The FY2026 priority review user fee for use of a tropical disease, rare paediatric disease or material threat MCM priority review voucher is $1,962,472, effective 1 October 2025 through 30 September 2026, payable in addition to any PDUFA fee.

    Fee Rate for Using a Priority Review Voucher in Fiscal Year 2026 · US Food and Drug Administration · Federal Register, doc. 2025-18075, 18 September 2025 2025
    What this evidence cannot show

    A correction notice to this rate was published 2026-07-28 (FR doc 2026-15147) and was not read; the corrected figure, if different, has not been verified here. The FY2027 rate (FR doc 2026-18464, published 2026-09-10) was not verified.

    Evidence that argues against this (1)
    • A fee, an exclusivity date or a programme status is a dated fact with a short shelf life: fee schedules are reset every fiscal year, exclusivity dates move on paediatric extension or litigation, and programme sunsets have been extended by Congress before. Anything planned on these has to re-query them. U.S. Food and Drug Administration 2026
  4. E4established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.paper

    FDA granted 569 rare paediatric disease designations between 2013 and 2022, of which 18% (n=105) were oncology, 26% neurology and 23% metabolism; details of rare paediatric disease designations are not publicly disclosable.

    Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development · Mease C, Miller KL, Fermaglich LJ, Best J, Liu G, Torjusen E · Orphanet Journal of Rare Diseases 19(1):86 2024
    PMID 38403586DOI
    What this evidence cannot show

    Authors are FDA Office of Orphan Products Development and Office of Pediatric Therapeutics staff analysing an internal, non-public FDA database, so the counts cannot be independently reproduced. A correction was published (PMID 38515155). Retrieved via PubMed.

    Evidence that argues against this (1)
    • 569 designations over ten years with an 18% oncology share is the published aggregate, and the entry states the limitation that undoes any finer reading: individual designations are not publicly disclosable. So the denominator is known and the composition cannot be checked, and no KMT2A-specific figure can be derived. U.S. Food and Drug Administration 2026
  5. E5strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.news

    Section 6604 of the Consolidated Appropriations Act, 2026, signed 3 February 2026 and carrying the Mikaela Naylon Give Kids a Chance Act, reauthorised the rare paediatric disease priority review voucher programme through 30 September 2029; the statute imposes no separate deadline for obtaining rare paediatric disease designation.

    Congress Extends Rare Pediatric Disease Priority Review Voucher Program Through 2029 · Spencer Fane LLP · Spencer Fane client insight 2026
    What this evidence cannot show

    Law-firm client alert, not the statutory text. Independently corroborated by Wilson Sonsini and Lowenstein Sandler alerts and by FDA's own programme page (E1), which is the agency source for the 2029-09-30 date. The underlying bill is H.R. 1262, 119th Congress.

    Evidence that argues against this (1)
    • Secondary commentary on section 6604 of the Consolidated Appropriations Act, 2026. The priority review voucher programme has lapsed and been reauthorised more than once, so any statement about its current status carries an implicit as-of date that a client alert does not always make explicit. Spencer Fane client insight 2026
  6. E6moderate confidence 3 of 5: One strong study, or several smaller consistent ones.news

    Reported priority review voucher transaction values range from $21.2m (2023) to $350m (AbbVie's 2015 purchase from United Therapeutics), with 2025-26 sales clustering at $150m-$200m including Jazz Pharmaceuticals at $200m in January 2026; approximately 63 rare paediatric disease vouchers have been awarded across 47 diseases.

    JPM26: Jazz inks $200M priority review voucher sale · Fierce Pharma · Fierce Pharma 2026
    What this evidence cannot show

    Trade press aggregating company disclosures. Individual prices are generally disclosed by the selling company in a press release or 8-K and are reliable; the aggregate counts (63 vouchers, 47 diseases) are journalistic and were not verified against an FDA list. Buyer identity is often undisclosed.

    Evidence that argues against this (1)
    • Trade-press reporting of transaction values is the weakest source class in this beat. Voucher sale prices are disclosed inconsistently — some in SEC filings, some in press releases, some only as analyst estimates — so a reported range mixes sources of very different reliability and the high and low ends may not be comparable quantities. Fierce Pharma 2026
  7. E7strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.news

    Abeona Therapeutics closed the sale of a rare paediatric disease priority review voucher for $155 million in 2025.

    Abeona Therapeutics Closes Sale of Rare Pediatric Disease Priority Review Voucher for $155 Million · Abeona Therapeutics Inc. · Abeona Therapeutics investor relations 2025
    What this evidence cannot show

    Company press release; it is a primary source for the company's own closed transaction, which is also a material disclosure, so the figure is reliable. Not an agency source.

    Evidence that argues against this (1)
    • A company press release announcing its own transaction is a primary record of the sale and the figure, and the corresponding SEC filing is the auditable version. For a single named transaction this is an adequate source; for the market-level range in E6 it is not. Abeona Therapeutics investor relations 2025
  8. E8strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.paper

    Across all 14 priority review vouchers awarded for neglected tropical disease products between 2007 and 2024, FDA approval came a median of 8.7 years after first regulatory approval or use in an endemic country and 5.2 years after inclusion in the WHO Essential Medicines List, suggesting the voucher programme has mainly rewarded regulatory filings for long-established therapies rather than stimulating innovation.

    Priority without progress: the FDA's neglected tropical disease voucher program after 18 years · Goh M, Outterson K, Kesselheim AS · Health Affairs Scholar 4(2):qxag024 2026
    PMID 41710508DOI
    What this evidence cannot show

    This analyses the neglected tropical disease arm, not the rare paediatric disease arm. The mechanism critique transfers (the voucher is awarded for an approval, not for new science), but the empirical finding does not automatically transfer to paediatric products. Retrieved via PubMed.

    Evidence that argues against this (1)
    • Mease et al. 2024 (E4) document 569 rare paediatric disease designations granted between 2013 and 2022 across a wide range of diseases, which the FDA authors read as evidence of genuinely broadened development. Note the authors are FDA Office of Orphan Products Development and Office of Pediatric Therapeutics staff working from a non-public internal database, so the count cannot be independently reproduced, and a correction was published as PMID 38515155. Mease C et al. 2024
  9. E9established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database

    FDA's Commissioner's National Priority Voucher pilot, announced in June 2025, targets a 1-2 month review against five declared national health priorities and issues vouchers that are explicitly nontransferable; the FDA programme page is marked content current as of 2026-08-26.

    Commissioner's National Priority Voucher (CNPV) Pilot Program · US Food and Drug Administration · FDA 2026
    What this evidence cannot show

    Retrieved 2026-09-18. Counts of vouchers awarded and named recipient products come from the same page and from trade press and were not independently checked. The pilot is discretionary and its statutory footing differs from the section 529 voucher.

    Evidence that argues against this (1)
    • A pilot announced in June 2025 with nontransferable vouchers is a programme whose operating rules and selection criteria are not yet established by practice. Nontransferability also removes the economic mechanism that makes the existing PRV valuable to a small sponsor, so it is not a substitute for the sunsetting programme. U.S. Food and Drug Administration 2026
  10. E10strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.database

    FDA's announcement of the 2024-11-15 revumenib approval for relapsed or refractory acute leukaemia with a KMT2A translocation records priority review and does not mention a rare paediatric disease designation or the award of a priority review voucher; it also notes that Revuforj for patients weighing less than 40 kg was initially available only through an expanded access programme because of delayed commercial availability.

    FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation · US Food and Drug Administration, Oncology Center of Excellence · FDA Resources Information Approved Drugs 2024
    What this evidence cannot show

    This is an absence-of-mention argument for the voucher. It is strong for the voucher (awards are public and FDA announces them) and weak for the designation (designations are not publicly disclosable per E4). The expanded-access detail was extracted by automated page summarisation and should be re-read verbatim before quoting.

    Evidence that argues against this (1)
    • An absence observed through a public search interface on one date bounds what that interface returns, not what exists. Regulatory documents are published on lags that differ by agency and by document type, a 404 can reflect a URL scheme change, and designations under several of these programmes are explicitly not publicly disclosable — so the absence is partly structural. U.S. Food and Drug Administration 2026
  11. E11strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.database

    FDA's announcement of the 2025-10-24 revumenib approval for relapsed or refractory NPM1-mutant AML in adult and paediatric patients 1 year and older records priority review and fast track designation and does not mention a rare paediatric disease designation or a priority review voucher.

    FDA approves revumenib for relapsed or refractory acute myeloid leukemia with a susceptible NPM1 mutation · US Food and Drug Administration, Oncology Center of Excellence · FDA Resources Information Approved Drugs 2025
    What this evidence cannot show

    Absence-of-mention argument, same limitation as E10.

    Evidence that argues against this (1)
    • An absence observed through a public search interface on one date bounds what that interface returns, not what exists. Regulatory documents are published on lags that differ by agency and by document type, a 404 can reflect a URL scheme change, and designations under several of these programmes are explicitly not publicly disclosable — so the absence is partly structural. Two announcements not mentioning a designation is also one source type counted twice. U.S. Food and Drug Administration 2026
  12. E12established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.trial_registry

    In the pivotal KMT2A-translocated cohort of SNDX-5613-0700 (AUGMENT-101, NCT04065399, n=104) the median age was 37 years (range 1 to 79) and 25 patients (24%) were under 17 years old.

    REVUFORJ (revumenib) tablets, for oral use - Full Prescribing Information, section 14.1, Table 10 · Syndax Pharmaceuticals, Inc. / US Food and Drug Administration · Drugs@FDA label NDA 218944 2024
    NCT04065399
    What this evidence cannot show

    Figures read directly from the approved label text. The label reports the age split at the 17-year boundary, not the 18-year boundary used in the statutory rare paediatric disease definition.

    Evidence that argues against this (1)
    • A median age of 37 in a study open from one year is the right fact for the commercial-gravity argument and it needs its complement stated: 25 of 104 patients (24%) were under 17 and the label records 13 infants. The distribution is not adult-only; it is adult-weighted, and the two support different conclusions about what a paediatric programme would have to add. Journal of Clinical Oncology 2025
Connected to