FDA Grants Priority Review to Alexion's Investigational Hypophosphatasia Drug
The U.S. Food and Drug Administration has accepted Alexion's Biologics License Application for investigational efzimfotase alfa (ALXN1850) for hypophosphatasia, granting it Priority Review. A decision is anticipated in the first half of 2027.
Alexion, AstraZeneca Rare Disease, has received a Priority Review designation from the U.S. Food and Drug Administration (FDA) for its Biologics License Application (BLA) for investigational efzimfotase alfa (ALXN1850). The therapy is being developed for the treatment of hypophosphatasia (HPP) in patients aged two years and older.
The FDA is expected to make a regulatory decision on the application during the first half of 2027. Priority Review is granted to applications for medicines that have the potential to offer significant improvements over available therapies in terms of safety, efficacy, or convenience.
Hypophosphatasia is a rare, inherited metabolic disorder characterized by deficient activity of alkaline phosphatase (ALP), an enzyme crucial for bone development and function. Efzimfotase alfa is an investigational enzyme replacement therapy designed for subcutaneous administration every two weeks, aiming to address the underlying enzyme deficiency.
The application is supported by Alexion's Phase III clinical program, including the HICKORY, MULBERRY, and CHESTNUT studies. These trials evaluated the therapy in treatment-naïve patients as well as those previously treated with Strensiq. Alexion estimates the addressable HPP population across key global markets to be approximately 13,800 patients.