AstraZeneca announced the U.S. Food and Drug Administration accepted and granted Priority Review for efzimfotase alfa. This investigational enzyme replacement therapy treats hypophosphatasia, a rare inherited metabolic disease that causes defective bone mineralization.
Priority Review status shortens the regulatory timeline for the Biologics License Application. The FDA expects to reach a regulatory decision in the first half of 2027.
If approved, efzimfotase alfa would be the first treatment for this condition designed for self-administration every two weeks. Data from three Phase III trials support the application.
AstraZeneca shares increased 0.4% in London trading following the announcement.