AstraZeneca's Rare Disease Drug Efzimfotase Alfa Granted FDA Priority Review

AstraZeneca's rare disease unit, Alexion, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application for efzimfotase alfa and granted it Priority Review. The drug is an investigational enzyme replacement therapy for treating hypophosphatasia (HPP), a rare, inherited metabolic disease that impairs bone and tooth development.

The Priority Review designation is granted to medicines that could offer significant improvements over existing treatments and shortens the FDA's review period, with a decision anticipated in the first half of 2027. This development is a crucial step for the HPP community and could bolster AstraZeneca's leadership in this rare disease space, potentially offering a new, less frequently dosed option compared to its existing HPP treatment, Strensiq.