A phase II basket trial of vosoritide in children with RASopathies, ACAN, and NPR2 deficiency
Authors' conclusion
Vosoritide led to marked increases in growth velocity in children with RASopathies, ACAN, and NPR2 deficiency, raising the possibility that vosoritide could be an effective precision medicine for all growth disorders affecting the MAPK pathway.
Study context
- Population
- Prepubertal children aged 3 to 11 years with a RASopathy, ACAN or NPR2 deficiency and height ≤ −2.25 SD
- Participants
- 30 children
- Exposure
- Vosoritide treatment vs prior observation period
- Outcome
- Co-primary: incidence of adverse events, change in annualized growth velocity (AGV), and height SD scores
- Follow-up
- 12-month treatment
Methodological detailssingle-arm, observation period followed by treatment period, single academic medical center
- Single-arm
- Observation period followed by treatment period
- Single academic medical center
Main finding
As reported in the abstract
AGV
Treatment vs ObservationHeight increase, SD (95% CI)P value
AGV
0.65(0.53–0.77)
P < 0.0001- Annualized growth velocity (AGV) increase P < .0001
- increase in age and sex-adjusted AGV Z-score 4.0 SD (95% CI 3.08–4.91); P < 0.0001
Observational data: associations, not effects.
Further findings
- The increase in AGV was seen in all genetic subgroups
Safety
- Discontinued medication due to adverse events with longer use
- 5 subjects
- Slipped capital femoral epiphyses
- 3
- Genu valgum
- 4 cases
- Short-term safety reassuring; mild injection site reactions most common adverse events
Original publication
A phase II basket trial of vosoritide in children with RASopathies, ACAN, and NPR2 deficiency. J Clin Endocrinol Metab. Published 10 April 2026. doi:10.
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