Clinical update


New guidance from the National Institute for Health and Care Excellence in agreement with NHS England, on accessing a potentially life-transforming drug for Hypophosphatasia.

Essential facts

Hypophosphatasia (HPP) is an extremely rare inherited bone condition that can appear any time from before birth to adulthood.

In babies it is often fatal, and in older children and adults it can be debilitating, leading to bone deformities. The disease is more common in older children and adults, affecting 1 in 6,370.

What’s new

More patients with HPP will access a potentially life-transforming drug due to a first of a kind agreement between NHS England and the manufacturer. The drug asfotase alfa was previously only recommended for use in babies by National Institute for Health and Care Excellence (NICE) in draft guidance, as evidence showed it could be life-saving. An agreement between NHS England and pharmaceutical company Alexion, alongside new draft NICE guidance, will broaden access


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