UK’s NICE supports restricted use of Alexion Pharmaceuticals’ Strensiq

The watchdog published draft guidance backing the drug's use in perinatal- and infantile-onset hypophosphatasia (HPP), a rare, and sometimes fatal, metabolic bone disease.

The guidance, however, do not endorse treatment for children with the juvenile-onset form of HPP.

The total cost per person per year of treatment is a whopping £367,000. But the draft guidance recommends Strensiq only as per a managed access agreement, under which the company offers the drug with the proposed cost cap, limiting the cost the NHS will have to pay per patient.

NICE health technology evaluation centre director Carole Longson said: “Based on the evidence presented by the company, as well as the testimony of clinical experts and patient representatives, the committee concluded that asfotase alfa improved the probability of survival in perinatal- and infantile-onset hypophosphatasia compared with best supportive care.

“The committee also accepted that asfotase alfa was likely to be clinically effective across a range of outcomes, such as reducing the need for respiratory support and the severity of rickets for people with perinatal- and infantile-onset hypophosphatasia.”

In October 2015, the US Food and Drug Administration approved Strensiq to treat patients with perinatal-, infantile- and juvenile-onset HPP.

HPP is characterized by low alkaline phosphatase activity and defective bone mineralization that can result in destruction and deformity of bones and other skeletal abnormalities, as well as systemic complications.

Strensiq replaces the missing TNSALP enzyme. In clinical studies of patients with HPP who had their first symptom prior to the age of 18, treatment with Strensiq enhanced overall survival in infants, improved bone mineralization, height, weight, and mobility.

Strensiq is approved in the US, European Union, Japan, and Canada.