AstraZeneca’s Efzimfotase Alfa Shows Promise in Rare Bone Disorder Trials

AstraZeneca has reported positive Phase III results for efzimfotase alfa, an investigational therapy for hypophosphatasia (HPP). The global programme included three trials—MULBERRY, CHESTNUT, and HICKORY—enrolling 196 patients across 22 countries, spanning children, adolescents, and adults.

Efzimfotase alfa is an enzyme replacement therapy designed to require lower injection volume and less frequent dosing compared to Strensiq. In the MULBERRY trial, involving children aged two to under 12, the drug met its primary endpoint, showing significant improvement in bone health at week 25. Secondary measures, including mobility and physical function, also supported its benefit.

The CHESTNUT study confirmed that patients switching from Strensiq maintained treatment benefits, while the HICKORY trial in older patients showed improvements in mobility and fatigue, though it did not meet its primary endpoint.

Overall, the therapy demonstrated an acceptable safety profile, highlighting its potential to improve treatment options for HPP patients.

01-04-2026