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Burosumab: Current status and future prospects.

Alpesh Goyal1, Nikhil Tandon1

  • 1Department of Endocrinology and Metabolism, All India Institute of Medical Sciences, Convergence Block, Room no 7002, Seventh Floor, New Delhi 110029, India.

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|October 20, 2023
PubMed
Summary
This summary is machine-generated.

Burosumab effectively treats hypophosphatemic rickets by targeting excess FGF23, improving phosphate levels and bone health. This therapy offers a novel approach beyond conventional treatments for this rare disease.

Keywords:
BurosumabFGF-23TIOXLHhypophosphatemiaosteomalaciarickets

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Area of Science:

  • Endocrinology
  • Nephrology
  • Skeletal Biology

Background:

  • Hypophosphatemic rickets/osteomalacia due to excess FGF23 is typically managed with phosphate and vitamin D, which address symptoms but not the underlying cause.
  • Conventional treatments can lead to complications like nephrocalcinosis and hyperparathyroidism, and often suffer from poor patient adherence.
  • Elevated Fibroblast Growth Factor 23 (FGF23) disrupts phosphate and vitamin D metabolism, causing significant skeletal abnormalities.

Purpose of the Study:

  • To evaluate the efficacy and safety of Burosumab, a monoclonal antibody targeting FGF23, in treating hypophosphatemic rickets and osteomalacia.
  • To assess Burosumab's impact on phosphate homeostasis, skeletal healing, and overall patient outcomes.
  • To provide an alternative therapeutic strategy for patients with FGF23-mediated hypophosphatemic rickets/osteomalacia.

Main Methods:

  • Burosumab, a monoclonal antibody, targets and neutralizes excess FGF23.
  • Clinical trials (Phase 2 and 3) were conducted to assess Burosumab's safety and efficacy.
  • Patient outcomes, including phosphate levels and rickets healing, were monitored.

Main Results:

  • Burosumab demonstrated overall safety and efficacy in clinical trials.
  • Treatment with Burosumab leads to improved phosphate homeostasis.
  • Healing of rickets and osteomalacia was observed in patients treated with Burosumab.
  • Burosumab is now FDA-approved for X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO).

Conclusions:

  • Burosumab offers a targeted therapy for FGF23 excess, addressing the root cause of hypophosphatemic rickets/osteomalacia.
  • Current expert recommendations suggest Burosumab for severe cases or those unresponsive to conventional therapy.
  • Further investigation into long-term data and cost-effectiveness is warranted for Burosumab.