NICE, Alexion Reach Agreement for NHS Access to Strensiq for Hypophosphatasia
NICE published draft guidance recommending Alexion Pharma’s Strensiq (asfotase alfa) for individuals with pediatric-onset hypophosphatasia, or HPP. The extremely rare inherited condition affects between one and seven babies each year in England.
A new agreement between Alexion and NHS England on the drug’s price, as well as a five-year managed access agreement, allowed NICE to widen the eligibility for treatment. Previous guidance recommended the treatment for babies with the most severe form.
The drug is the first therapy that specifically targets the underlying cause of HPP. The managed access agreement means Strensiq will be made available to eligible children and adults for five years while information is gathered on how long treatment should continue.