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Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia.

Publication ,  Journal Article
Weber, TJ; Imel, EA; Carpenter, TO; Peacock, M; Portale, AA; Hetzer, J; Merritt, JL; Insogna, K
Published in: J Clin Endocrinol Metab
December 17, 2022

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.

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Published In

J Clin Endocrinol Metab

DOI

EISSN

1945-7197

Publication Date

December 17, 2022

Volume

108

Issue

1

Start / End Page

155 / 165

Location

United States

Related Subject Headings

  • Phosphates
  • Humans
  • Genetic Diseases, X-Linked
  • Familial Hypophosphatemic Rickets
  • Endocrinology & Metabolism
  • Antibodies, Monoclonal, Humanized
  • Adult
  • 1114 Paediatrics and Reproductive Medicine
  • 1103 Clinical Sciences
 

Citation

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Weber, T. J., Imel, E. A., Carpenter, T. O., Peacock, M., Portale, A. A., Hetzer, J., … Insogna, K. (2022). Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. J Clin Endocrinol Metab, 108(1), 155–165. https://doi.org/10.1210/clinem/dgac518
Weber, Thomas J., Erik A. Imel, Thomas O. Carpenter, Munro Peacock, Anthony A. Portale, Joel Hetzer, J Lawrence Merritt, and Karl Insogna. “Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia.J Clin Endocrinol Metab 108, no. 1 (December 17, 2022): 155–65. https://doi.org/10.1210/clinem/dgac518.
Weber TJ, Imel EA, Carpenter TO, Peacock M, Portale AA, Hetzer J, et al. Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Dec 17;108(1):155–65.
Weber, Thomas J., et al. “Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia.J Clin Endocrinol Metab, vol. 108, no. 1, Dec. 2022, pp. 155–65. Pubmed, doi:10.1210/clinem/dgac518.
Weber TJ, Imel EA, Carpenter TO, Peacock M, Portale AA, Hetzer J, Merritt JL, Insogna K. Long-term Burosumab Administration Is Safe and Effective in Adults With X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Dec 17;108(1):155–165.
Journal cover image

Published In

J Clin Endocrinol Metab

DOI

EISSN

1945-7197

Publication Date

December 17, 2022

Volume

108

Issue

1

Start / End Page

155 / 165

Location

United States

Related Subject Headings

  • Phosphates
  • Humans
  • Genetic Diseases, X-Linked
  • Familial Hypophosphatemic Rickets
  • Endocrinology & Metabolism
  • Antibodies, Monoclonal, Humanized
  • Adult
  • 1114 Paediatrics and Reproductive Medicine
  • 1103 Clinical Sciences