NICE extends recommendation for Strensiq (asfotase alfa) to treat people with paediatric-onset hypophosphatasia.- Alexion Pharma

NICE has published draft guidance recommending the drug asfotase alfa (also called Strensiq and made by Alexion Pharma UK) for people with paediatric-onset hypophosphatasia. The extremely rare inherited condition affects between 1 and 7 babies each year in England.

A new agreement between the company and NHS England on price and access to the drug means NICE can now widen the group of people eligible for treatment on the NHS. Previous NICE draft guidance recommended the treatment for only babies with the most severe form.

Now an improved deal includes a 5-year managed access agreement which reduces the cost of the drug to the NHS and enables people with the highest unmet need to be identified. Described as a �step-change� in the management of the condition, the drug offers a �lifeline� for people that could enable them to have a good quality of life but without which they would not survive. The drug is the first therapy that specifically targets the underlying cause of hypophosphatasia.