NICE backs Strensiq for wider indication following MAA

Medicines cost-effectiveness watchdog the National Institute for Health and Care Excellence (NICE) today published final draft guidance recommending asfotase alpha for people with pediatric-onset hypophosphatasia – a very rare inherited condition affecting between one and seven babies each year in England.

The draft guidance changes the NICE’s previous draft by recommending US firm Alexion Pharmaceuticals (Nasdaq: ALXN) Strensiq (asfotase alfa) for the wider population of people with the condition, not just babies with the most severe form. This follows an improved deal, including a five-year managed access agreement (MAA), between NHS England and the company which reduces the cost of the drug to the NHS and enables people with the highest unmet need to be identified. Further

The MAA has been developed in collaboration between physician thought-leaders, patient groups, NHS England, and Alexion. The MAA ensures access to Strensiq for infants, children and adult patients with pediatric-onset HPP who experience the most disabling symptoms and are expected to benefit most from therapy.