GlobalResearch and clinical trials
Efzimfotase alfa in hypophosphatasia: positive in children, main goal not met in adolescents and adults
An early result that has not yet been published in a peer-reviewed journal, or comes from a small, uncontrolled study. Treat it with caution.
The phase 3 program included 196 patients from 22 countries. In the trial with 29 children, bone health improved markedly compared with placebo. In 43 children who had been treated before, the benefit continued. In 124 adolescents and adults, however, the main goal on a walking test was not reached.
What does it mean for patients and families?
The results come from a company announcement and are not yet a peer-reviewed publication. The drug is not approved. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Hypophosphatasia (HPP)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.