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Crysvita (BUROSUMAB)
Burosumab-twza binds to and inhibits FGF23, restoring renal phosphate reabsorption and increasing 1,25 dihydroxy vitamin D levels.
Crysvita (Burosumab) is a small molecule modality developed by Ultragenyx Pharmaceuticals for the treatment of Familial X-linked Hypophosphatemic Vitamin D Refractory Rickets and Osteomalacia. It targets fibroblast growth factor 23, a protein involved in phosphate regulation. Crysvita was FDA-approved in 2018 and remains a patented product. Key safety considerations include monitoring of serum phosphate levels and potential effects on kidney function. It is a relatively new treatment option for these rare conditions.
At a glance
| Generic name | BUROSUMAB |
|---|---|
| Sponsor | Ultragenyx Pharm Inc |
| Target | FGF23 |
| Modality | Monoclonal antibody |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
| First approval | 2018 |
| Annual revenue | 700 |
Mechanism of action
X-linked hypophosphatemia results from excessive FGF23, which reduces phosphate reabsorption in the kidneys and decreases 1,25 dihydroxy vitamin D production. Burosumab-twza targets and neutralizes FGF23, thereby improving phosphate reabsorption and boosting 1,25 dihydroxy vitamin D levels.
Approved indications
- Familial x-linked hypophosphatemic vitamin D refractory rickets
- Osteomalacia
Common side effects
- Pyrexia
- Injection site reaction
- Cough
- Vomiting
- Pain in extremity
- Headache
- Tooth abscess
- Dental caries
- Diarrhea
- Vitamin decreased
- Rash
- Toothache
Key clinical trials
- X-linked Hypophosphatemia Disease Monitoring Program
- Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
- Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia (PHASE2)
- Tumor-induced Osteomalacia Disease Monitoring Program
- Effective Dosing of Burosumab in XLH
- Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients
- Expanded Access to Burosumab
- Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age (PHASE1,PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| SEC EDGAR | Revenue + earnings |