What the labels measure in a treated child: bone growth, in one sentence, and nothing else endocrine
also called paediatric endocrine monitoring menin inhibitor · REVUFORJ 8.4 pediatric use · children menin inhibitor endpoints
One menin drug is approved for children from one year old and has been given to 132 of them, including 25 babies. The only endocrine or growth instruction in its label is a single sentence telling doctors to monitor bone growth and development. Nothing says how, how often, or with what test.
The same thing, for a scientist
Read directly from both DailyMed labels on 2026-09-19 [E1][E2]. **REVUFORJ**: safety and efficacy established in patients 1 year and older; the paediatric experience comprises **25 infants under 2 years, 78 children aged 2 to under 12 and 29 adolescents aged 12 to under 17** - 132 children in total; dosing below 40 kg is BSA-based; safety and effectiveness below 1 year of age are not established. The only paediatric-specific instruction of any endocrine or developmental kind is: *"Based on the findings in animals, monitor bone growth and development in pediatric patients"* - prompted by irreversible femoral growth-plate closure in 6-7-week-old rats at ~2x the human AUC. **KOMZIFTI**: *"Safety and effectiveness of KOMZIFTI in pediatric patients have not been established."* Nothing further. Neither label specifies a measurement, an interval, a threshold or an action for the bone-growth instruction, and neither mentions height velocity, bone age, pubertal staging, IGF-1, thyroid function, gonadotrophins or glucose anywhere.
The instrumentation, itemised
| Domain | Measured? | Where |
|---|---|---|
| Bone growth and development | Instructed, unspecified | REVUFORJ 8.4, one sentence 1 |
| Intact PTH | Measured and reported (34%) | REVUFORJ 6.1, pooled adults + children 1 |
| Calcium, phosphate, ALP | Measured and reported | REVUFORJ 6.1 1; ziftomenib reports ALP only 2 |
| Height, weight velocity, growth charts | No | - |
| Bone age radiograph | No | - |
| Pubertal (Tanner) staging | No | - |
| IGF-1, growth hormone axis | No | - |
| Thyroid function | No | - |
| Gonadotrophins, gonadal function | No (despite irreversible animal fertility findings) 2 | - |
| Glucose, insulin, HbA1c | No | see concept-not-one-glucose-value-appears-on-either-menin-inhibitor-label |
| Bone density (DXA) | No | - |
| Any paediatric subgroup analysis of the endocrine labs | No - children are pooled with adults | 1 |
The pooling problem
The 34% intact-PTH figure is drawn from a pooled population of 241 patients, 207 adults and 34 children in the laboratory-table population 1. No paediatric-specific rate is given for any endocrine laboratory abnormality. A child's parathyroid, mineral axis and bone are not an adult's, and the one population in which the growth-plate finding is actionable is the one whose data are not separated out. This is a reporting decision, not a data limitation - the sponsor can stratify by age in an afternoon.
How the one instruction compares with paediatric practice elsewhere
Drugs with recognised effects on growth in children - inhaled and systemic corticosteroids, stimulants, growth-hormone-axis agents - carry specified monitoring: serial height on a growth chart at defined intervals, with an action threshold. The REVUFORJ instruction names no measurement and no interval, so compliance with it is unfalsifiable and its outputs are not collected anywhere centrally. Combined with concept-endocrine-endpoint-absence-menin-trial-registries (zero endocrine outcome measures across AUGMENT-101, KOMET-001 and cAMeLot-1) and FDA's own PMR 4730-1, which requires a chronic-exposure endocrine trial but names no paediatric endpoint, the result is that the population with the most to lose is the one generating the least data.
| revuforj paediatric n | 132 |
|---|---|
| revuforj infants under 2 | 25 |
| revuforj children 2 to 12 | 78 |
| revuforj adolescents 12 to 17 | 29 |
| minimum age | 1 year |
| dosing under 40kg | BSA-based |
| only paediatric endocrine instruction | monitor bone growth and development (unspecified) |
| paediatric subgroup endocrine labs | not reported |
| ziftomenib paediatric | not established |
- The only paediatric-specific safety instruction in the class names no measurement and no interval, so it generates no data and cannot be audited.
- Pooling 34 children with 207 adults hides exactly the subgroup in whom the growth-plate finding is actionable.
- Irreversible animal fertility findings sit on a label for a class given to children, with no reproductive endpoint collected anywhere.
- What are the endocrine laboratory rates in the 34 paediatric patients specifically? This is a stratification of data that already exists.
- Has any treating centre collected serial heights on menin-inhibitor-treated children, and would a consortium retrospective assemble a first growth dataset?
- Does the FDA multi-discipline review for NDA 218944 contain paediatric endocrine subgroup data that did not reach the label?
- E1established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database
REVUFORJ is approved from 1 year of age with 132 children treated (25 infants <2y, 78 aged 2-<12y, 29 aged 12-<17y); its only paediatric endocrine/developmental instruction is to monitor bone growth and development, unspecified; endocrine laboratory abnormalities are reported only pooled with adults.
REVUFORJ (revumenib) tablets, for oral use - US Prescribing Information (SPL version 5, published 20 Feb 2026) · Syndax Pharmaceuticals, Inc. · DailyMed / FDA Structured Product Label, setid 6eb3cdbc-0e74-477d-82d6-3bb172d3f63f 2026What this evidence cannot show
Read from the DailyMed SPL text on 2026-09-19. The label is a summary document; the FDA multi-discipline review may contain paediatric subgroup data not reproduced on the label, and has not been read for this entry.
Evidence that argues against this (1)
- FDA postmarketing requirement 4730-1 does compel a chronic-exposure trial addressing endocrine-related disorders due to chronic menin inhibition, so the regulator has acted even though the label does not specify paediatric endpoints - see reg-fda-pmr-endocrine-chronic-menin-inhibition. Syndax Pharmaceuticals et al. 2026
- E2established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database
KOMZIFTI states that safety and effectiveness in paediatric patients have not been established, and reports irreversible animal fertility findings after a 4-week recovery period.
KOMZIFTI (ziftomenib) capsules, for oral use - US Prescribing Information (SPL version 1, published 16 Dec 2025) · Kura Oncology, Inc. · DailyMed / FDA Structured Product Label, setid b650f696-3391-4274-8b55-a5f5e9d04769 2025What this evidence cannot show
A 'not established' statement reflects the absence of a paediatric development programme at approval rather than a negative finding; a paediatric programme may follow.
Evidence that argues against this (1)
- Absence of a paediatric indication also means no children are exposed to ziftomenib on label, so the missing paediatric monitoring is currently a hypothetical rather than an active exposure for this product. Kura Oncology et al. 2025
The growth-plate finding produced exactly one label sentence, with no named measurement, interval or action threshold
Itemises exactly what the proposed surveillance panel would add, and confirms that none of it is currently required: no height velocity, bone age, Tanner staging, IGF-1, thyroid, gonadotrophin, glucose or DXA
The PMR compels a chronic-exposure endocrine trial but names no paediatric endpoint, so the population with the most to lose generates the least data
Irreversible animal fertility findings and skeletal teratogenicity below clinical exposure, with no reproductive or craniofacial endpoint collected in any treated child
Model M2 would produce the paediatric stratification whose absence that entry documents