Burosumab beats conventional therapy for rare bone disorder in real-world study

Burosumab beats conventional therapy for rare bone disorder in real-world study

A real-world study published in the Journal of Bone and Mineral Research confirms that burosumab-twza (Crysvita) significantly outperforms conventional therapy — vitamin D alone or combined with oral phosphate — for adults living with X-linked hypophosphatemia (XLH), a rare inherited form of rickets. The FDA first approved burosumab-twza in 2018, and this new data from 109 adults in the XLH disease monitoring program adds important long-term, real-world evidence to support its use.

Over three years, patients on burosumab showed sustained improvements in serum phosphate and meaningful reductions in pain, stiffness, and physical impairment — benefits that grew more pronounced over time. While year-one patient-reported improvements weren't statistically significant, by year three the differences were clear, suggesting the drug's effect builds with continued use.

By the Numbers:

• Serum phosphate increase at 3 years: +0.6 mg/dL (burosumab) vs. +0.1 mg/dL (conventional therapy)
• 60.8% of burosumab patients reached normal phosphate levels at 3 years vs. 25% on conventional therapy
• Pain score improvement: 9.8 points vs. 3.3 points in favor of burosumab

Why it matters: For patients with XLH — a condition that causes chronic pain, skeletal deformities, and functional limitations — these real-world findings reinforce clinical guidelines supporting burosumab-twza as the preferred treatment option, offering meaningful hope for better long-term quality of life.