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The U.K.'s NICE issued draft guidance recommending approval of Strensiq asfotase alfa from Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) to treat the bone manifestations of perinatal- and infantile-onset hypophosphatasia as part of a managed access agreement.
The recommendation covers patients whose onset of the disease occurs before six months of age. The drug is approved in Europe for that population as well as juvenile-onset HPP, which includes patients with disease onset prior to 18 years of age.
The recommendation is contingent on Alexion providing Strensiq under a discount scheme that includes an undisclosed maximum cost cap per patient. Alexion is also to "provide further opportunities to reduce the short-term cost" of Strensiq to the NHS, the guidance said.
Strensiq is a fusion protein incorporating the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP; ALPL) and a bone-targeting peptide. According to NICE, its cost varies from L165,000 ($215,457) for a baby to L1.5 million ($2 million) for an 80 kg adult, based on its list price. Comments on the draft guidance are due Oct. 13.
In December guidance, NICE recommended against Strensiq's use. At the time, the committee had said there was "considerable uncertainty around the robustness" of clinical results and natural history data, as well as the "precise size" of the drug's benefit.
Alexion gained $3.33 to $133.69 on Thursday.
Original Article: NICE backs Strensiq for some HPP patientsNEXT ARTICLE
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