Drug updated on 3/28/2024
| Dosage Form | Injectable (subcutaneous; 10 mg/mL, 20 mg/mL, 30 mg/mL) |
| Drug Class | Fibroblast growth factor blocking antibodies |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.
- 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.
Summary
- Burosumab (Crysvita) is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older, as well as 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.
- A total of three systematic reviews/meta-analyses were reviewed which provided insights into the efficacy, safety profile, clinical characteristics, prevalence rates, diagnosis delays, bone density impacts related to burosumab use.
- In a meta-analysis involving randomized controlled trials (RCTs), it was found that compared to control groups serum phosphorus level significantly increased within the burosumab group; this increase also validated by changes observed in secondary events.
- The safety profile comparison between RCTs' control group versus those treated with burosumab showed no significant difference; however single-arm combined group data demonstrated high incidence rate for any treatment emergency adverse event but most adverse events severity ranged from mild to moderate while serious TEAE rate remained low.
- Tumor-induced osteomalacia cases are more frequent among adult men who show higher fracture incidences than women; promising non-surgical therapies include treatments using Crysvita especially when elevated fibroblast growth factor-23 levels are present or radiolabeled somatostatin analogs if neoplasm can be identified via somatostatin receptor-based imaging techniques.
- Persistent or recurrent cases of TIO remain either undiagnosed due to tumor localization issues or persist after surgery making these active disease patients potential candidates for Crysvita treatment given its ability to manage symptoms such as fragility fractures/pseudofractures along with disabling bone/muscle pain.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Crysvita (burosumab-twza) Prescribing Information. | 2020 | Ultragenyx Pharmaceutical Inc., Novato, CA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy and safety of burosumab in X-linked hypophosphataemia: Systematic review and meta-analysis. | 2023 | The journal of endocrinology and metabolism |
| Tumor-induced Osteomalacia: A Systematic Review and Individual Patient's Data Analysis. | 2022 | The journal of 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 review report: burosumab (Crysvita). | 2020 | CADTH |