Researchers have presented data from a Phase 2 study of Alexion Pharmaceuticals' (ALXN) targeted enzyme replacement therapy asfotase alfa in children and infants with hypophosphatasia.
The study met its primary endpoint of improving skeletal mineralization from baseline.
Improvement was observed as soon as 12 weeks and continued at 48 weeks, with 93% of those treated surviving the first four years of treatment.
The drug has a Breakthrough Therapy designation. (PR)