18th Sep 2026 08:42
(Alliance News) - AstraZeneca PLC on Friday said the US Food & Drug Administration granted a 'priority review' designation for efzimfotase alfa, an investigational enzyme replacement therapy.
The Cambridge, England-based pharmaceutical company said the US Food & Drug Administration has accepted and granted 'priority review' designation to Alexion's biologics licence application for efzimfotase alfa to treat patients aged two years and older with hypophosphatasia, a rare inherited metabolic disease.
AstraZeneca explained hypophosphatasia "is characterised by defective mineralisation [the process that hardens and strengthens bones and teeth], impaired calcium and phosphate regulation and functional impairments, such as muscle weakness, neurologic symptoms, generalised fatigue and pain that can be debilitating."
The disease is caused by caused by deficient activity of the enzyme alkaline phosphatase [ALP], which is important for building healthy bones and supporting proper muscle function, AstraZeneca said.
The FDA's regulatory decision is expected in the first half of 2027.
The application is supported by results from the phase 3 Hickory, Mulberry and Chestnut trials, in which AstraZeneca said efzimfotase alfa demonstrates a favourable safety profile and is generally well tolerated.
If approved, the drug would be the first treatment designed to address skeletal abnormalities and functional impairments regardless of disease onset, with self-administration every two weeks.
Regulatory submissions are also under review in Japan and other markets.
AstraZeneca shares were 0.4% higher at 12,478.00 pence each on Friday morning in London.
By Tom Budszus, Alliance News slot editor
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