Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia.
CONTEXT: Burosumab was developed as a treatment option for patients with the rare, lifelong, chronically debilitating, genetic bone disease X-linked hypophosphatemia (XLH). OBJECTIVE: Collect additional information on the safety, immunogenicity, and clinical response to long-term administration of burosumab. METHODS: UX023-CL203 (NCT02312687) was a Phase 2b, open-label, single-arm, long-term extension study of adult subjects with XLH who participated in KRN23-INT-001 or KRN23-INT-002 studies. The long-term UX023-CL203 study (January 5, 2015 through November 30, 2018) provided data up to 184 weeks. Participants in UX023-CL203 received burosumab based on the last dose in the prior KRN23-INT-001 or KRN23-INT-002 studies (0.3, 0.6, or 1.0 mg/kg given by subcutaneous injection every 4 weeks). At Week 12, burosumab could be titrated upward/downward to achieve fasting serum phosphate levels within the normal range. Primary objectives included long-term safety, the proportion of subjects achieving fasting serum phosphate in the normal range, changes in bone turnover markers, patient-reported outcomes for pain and stiffness, and measures of mobility. RESULTS: Fasting serum phosphate levels at the midpoint of the dosing interval (2 weeks postdose, the time of peak effect) were within the normal range in 85% to 100% of subjects. Measures of phosphate metabolism and bone biomarkers generally improved with burosumab therapy, approaching or reaching their respective normal ranges by study end. Improvements in patient-reported outcomes and mobility were sustained throughout the observation period. No new safety findings emerged with longer-term burosumab treatment. CONCLUSION: These data support the conclusion that burosumab therapy may be a safe and effective long-term treatment option for adult patients with XLH.
Duke Scholars
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- Phosphates
- Humans
- Genetic Diseases, X-Linked
- Familial Hypophosphatemic Rickets
- Endocrinology & Metabolism
- Antibodies, Monoclonal, Humanized
- Adult
- 3202 Clinical sciences
- 1114 Paediatrics and Reproductive Medicine
- 1103 Clinical Sciences
Citation
Published In
DOI
EISSN
Publication Date
Volume
Issue
Start / End Page
Location
Related Subject Headings
- Phosphates
- Humans
- Genetic Diseases, X-Linked
- Familial Hypophosphatemic Rickets
- Endocrinology & Metabolism
- Antibodies, Monoclonal, Humanized
- Adult
- 3202 Clinical sciences
- 1114 Paediatrics and Reproductive Medicine
- 1103 Clinical Sciences