Source: Sharecast
Efzimfotase alfa is an investigational enzyme replacement therapy for people with hypophosphatasia, a rare, inherited metabolic disease that softens bones and causes patients to lose teeth.
One phase III trial did not achieve statistical significance in the primary endpoint in treatment-naïve adolescents and adults, AstraZeneca confirmed. Treatment-naïve patients have yet to receive any form of treatment.
But another trial showed a "statistically significant and clinically meaningful improvement" in bone health in treatment-naïve paediatric patients, while a second demonstrated safety and tolerability in children previously treated with another AstraZeneca drug, Strensiq.
Efzimfotase alfa is designed to offer lower injection volume and less frequent dosing that Strensiq.
Marc Dunoyer, chief executive of Alexion, AstraZeneca Rare Disease, said: "We are encouraged by the improvements observed across this patient population who exhibit a wide range of severity and clinical characteristics.
"Collectively, these results support the potential for efzimfotase alfa to transform the treatment paradigm for people living with this rare disease."
A total of 196 children, adolescents and adults in 22 countries were enrolled in the global phase III clinical programme, with either paediatric-onset or adult-onset hypophosphatasia.
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