Clinical trial results of Strensiq® treatment of patients with hypophosphatasia were demonstrated over both the short- and long-term.
Mean alkaline phosphatase levels varied across populations studied in Strensiq clinical trials and increased typically well above the upper limit of normal.8
In patients with perinatal/infantile-onset hypophosphatasia, Strensiq improved survival and invasive ventilation-free survival at 1 year (week 48) vs historical controls.1,a
74% (50/68) of patients with perinatal/infantile-onset hypophosphatasia and 100% (8/8) of patients with juvenile-onset hypophosphatasia achieved substantial or complete healing of hypophosphatasia-related rickets at the mean time interval of 24 months and by month 54 of treatment, respectively.1,8,b
Strensiq gradually increased height and weight z scores over the course of treatment in patients with perinatal/infantile- and juvenile-onset hypophosphatasia.1,c
Strensiq improved mobility and gait in patients with juvenile-onset hypophosphatasia. 100% (6/6) of patients with juvenile-onset hypophosphatasia had percent-predicted 6MWT values within the normal range for age-, sex-, and height-matched peers at 48 months after starting treatment.1,d
aOverall and invasive ventilation-free survival were assessed in Studies 1 and 2.
bAs measured by a score of ≥2 on the RGI-C scale in Studies 1, 2, and 3.
cGrowth was assessed in Studies 1, 2, and 3 using height and weight z scores.
dMobility and gait assessments were conducted in Study 3 using 6MWT and MPOMA-G.