Efzimfotase alfa for treating hypophosphatasia

Efzimfotase alfa is currently in clinical development for hypophosphatasia (HPP). HPP is a rare, highly variable genetic condition that features abnormalities in the bone and/or tooth mineralisation, caused by a deficiency of an enzyme called tissue-nonspecific alkaline phosphatase (TNSALP). This plays a key role in creating and maintaining healthy bones and managing calcium and phosphate in the body. The reduction of this enzyme’s activity leads to ricket-like symptoms, softening and weakening of the bones and a greater incidence of fractures. The signs and symptoms of HPP vary widely and can appear anytime from before birth to adulthood. Currently, there is only one approved treatment for HPP for individuals who develop the condition at an early age before adulthood, which requires frequent dosing. Therefore, there is a need for alternative treatment options for HPP to expand patient choice.