1 March 2023 - This evaluation reviews the evidence for asfotase alfa for the treatment of patients with paediatric‑onset hypophosphatasia (HST6), including evidence collected as part of the managed access agreement.
Asfotase alfa is recommended as an option for the treatment of patients with paediatric‑onset hypophosphatasia if the person's symptoms started before or at birth (perinatal onset) or between the ages of 0 and 6 months (infantile onset).
It is also recommended for certain patients whose symptoms started between the ages of 6 months and 17 years (juvenile onset).