Safety, tolerability, pharmacokinetics, and efficacy of burosumab in infants with X-linked hypophosphataemia: an open-label, multicentre, non-randomised study
Authors' conclusion
This study confirms the safety and tolerability of burosumab in patients with XLH initiating treatment aged less than 12 months and is consistent with previous reports with no new safety concerns.
Study context
- Population
- Infants aged <12 months with X-linked hypophosphataemia (XLH) (pathogenic, likely pathogenic or uncertain-importance PHEX variant in participant or directly related family member) and hypophosphataemia; sites in Austria, France, Italy, Spain and the UK15 completed
- Participants
- 16 infants
- Comparison
- Cohorts 1 and 2 vs cohort 3
- Cohort 3n = four
- Primary endpoint
- Safety and tolerability, focus on TEAEs, in the full-analysis set
- Follow-up
- Up to 48 weeks
Methodological detailsopen-label, multicentre, phase 1/2, Recruitment Feb 26, 2020; completed March 6, 2024
- Open-label
- Multicentre
- Phase 1/2
- Recruitment Feb 26, 2020; completed March 6, 2024
Primary result
As reported in the abstract
- Pyrexia (most common TEAE)
- 12 (75%) of 16 (95% CI 47.6-92.7)
- Participants with ≥1 treatment-related TEAE
- four
- Blood PTH increase (most common treatment-related TEAE)
- three (19%) of 16 (4.0-45.6)
- Developed antidrug antibodies
- one
Safety
- All participants had treatment-emergent adverse events (TEAEs); ≥1 treatment-related TEAE
- 4
- Pyrexia
- 12 (75%) of 16
- Blood PTH increase (treatment-related)
- three (19%) of 16
- No TEAEs led to treatment discontinuation or death
Original publication
Safety, tolerability, pharmacokinetics, and efficacy of burosumab in infants with X-linked hypophosphataemia: an open-label, multicentre, non-randomised study. Lancet Diabetes Endocrinol. Published 24 April 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →