Drug updated on 12/11/2024
| Dosage Form | Injection (subcutaneous; 10 mg/mL, 20 mg/mL, 30 mg/mL) |
| Drug Class | Fibroblast growth factor 23 (FGF23) blocking antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older
- Indicated for the treatment of FGF23-related hypophosphatemia in tumor induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older.
Latest News
Summary
- This summary is based on the review of four systematic review(s)/meta-analysis(es). [1-4]
- In pediatric patients with X-linked hypophosphatemia (XLH), burosumab significantly improved biochemical markers (1,25-dihydroxyvitamin D, phosphorus, alkaline phosphatase), renal function (TmP/GFR), and skeletal health, as shown by improvements in the rickets severity score (RSS) and the 6-minute walk test (6MWT).
- Compared to conventional therapy (phosphorus and calcitriol), burosumab was more effective in normalizing phosphate homeostasis and reducing skeletal lesions, including a significant reduction in Thacher's total rickets severity score (standardized mean difference (SMD): -1.46, 95% CI: -1.76 to -1.17, p < 0.001) and a decline in serum alkaline phosphatase levels (SMD: 130.68, 95% CI: 125.26-136.1, p < 0.001).
- In adults with XLH, burosumab normalized phosphorus levels (SMD: 1.23, 95% CI: 0.98-1.47, p < 0.001) and led to clinical improvement, while also showing potential as a nonsurgical treatment option for tumor-induced osteomalacia (TIO), particularly in cases of persistent or recurrent disease.
- Burosumab was well-tolerated in children with XLH, with only mild treatment-related adverse effects reported, though long-term safety data, particularly regarding height and quality of life, remain to be determined.
- No specific adverse effects were detailed for TIO patients receiving burosumab, and the safety profile in adults with XLH and TIO was generally positive.
- Burosumab demonstrated significant effectiveness in pediatric patients with XLH by improving biochemical markers, renal function, and skeletal health, while in adults with XLH, it normalized phosphorus levels and led to clinical improvement. For TIO, burosumab showed promise as a nonsurgical treatment, particularly for patients with persistent or recurrent disease.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Crysvita (burosumab-twza) Prescribing Information. | 2023 | Kyowa Kirin, Inc., Princeton, NJ |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Meta-analysis and systematic review: burosumab as a promising treatment for children with X-linked hypophosphatemia | 2024 | Frontiers in Endocrinology |
| Efficacy and safety of burosumab compared with conventional therapy in patients with X-linked hypophosphatemia: A systematic review | 2024 | Archives of Endocrinology and Metabolism |
| Tumor-induced Osteomalacia: A Systematic Review and Individual Patient's Data Analysis | 2022 | The Journal of Clinical Endocrinology and Metabolism |
| Persistence and recurrence in tumor-induced osteomalacia: A systematic review of the literature and results from a national survey/case series | 2022 | Endocrine |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Asia-pacific consensus recommendations on x-linked hypophosphatemia: diagnosis, multidisciplinary management, and transition from pediatric to adult care. | 2023 | Journal of Bone and Mineral Research Plus |
| Clinical practice recommendations for the diagnosis and treatment of X-linked hypophosphatemia: a consensus based on the ADAPTE method. | 2022 | Medicina Clinica |
| Clinical practice guidelines for paediatric X-linked hypophosphataemia in the era of burosumab. | 2022 | Journal of Paediatrics and Child Health |