Second interim analysis of the post-authorisation safety study (PASS) of burosumab in paediatric patients with X-linked hypophosphataemia
Authors' conclusion
The safety profile of burosumab was consistent with previous studies, and no new safety concerns were reported for children or adolescents.
Study context
- Population
- Children and adolescents with X-linked hypophosphataemia (XLH) in the International X-linked Hypophosphataemia Registry
- Participants
- 340 vs 91
- Exposure
- Burosumab vs phosphate and/or active vitamin D
- Burosumabn = 340
- Phosphate and/or active vitamin Dn = 91reference
- Outcome
- Safety outcomes in children and adolescents with XLH
- Follow-up
- 5 years after study initiation
Methodological detailsInternational X-linked Hypophosphataemia Registry, prespecified interim analysis
- International X-linked Hypophosphataemia Registry
- Prespecified interim analysis
Main finding
As reported in the abstract
≥1 adverse event
- Events possibly/probably related to burosumab
- 49 (14.4%)
- Events leading to burosumab withdrawal
- 0
- Treatment-related serious adverse events, both cohorts
- 0
- Deaths
- 0
- Hospitalisations
- ∼71%
Observational data: associations, not effects.
Safety
Adverse events
- Adverse events — Possibly/probably burosumab-related events
- 49 (14.4%)
Adverse events
- No treatment-related serious adverse events and no deaths in either cohort
Original publication
Second interim analysis of the post-authorisation safety study (PASS) of burosumab in paediatric patients with X-linked hypophosphataemia. Eur J Endocrinol. Published 23 June 2026. doi:10.
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