On Sunday, AstraZeneca Plc (NYSE:AZN) reported positive late-stage data for investigational therapy efzimfotase alfa in children with hypophosphatasia (HPP), while separately securing European Union approval for cancer drug Enhertu in a broad group of HER2-positive solid tumors, marking two significant milestones across its rare disease and oncology portfolios.
Efzimfotase Alfa Meets Primary And Secondary Endpoints In HPP Trial
The company said data from the Phase 3 MULBERRY trial showed efzimfotase alfa, also known as ALXN1850, significantly improved bone health in children aged 2 to under 12 years with HPP who had not previously received Strensiq (asfotase alfa).
Patients treated with efzimfotase alfa achieved a median Radiographic Global Impression of Change (RGI-C) score of 1.67 at week 25, compared with 0 in the placebo group, representing a statistically significant difference (p=0.0003).
The trial also met its key secondary endpoint, with patients receiving the therapy showing significant improvement in Rickets Severity Score versus placebo.
