NICE backtracks on its 'no' for Strensiq, but Alexion still isn't satisfied

England’s cost watchdogs are changing their tune on Alexion’s ($ALXN) rare bone disorder drug Strensiq--but not quite as much as the drugmaker would like.

Thursday, the National Institute of Health and Care Excellence (NICE) backed the once-rejected med for hypophosphatasia patients whose disease began by the time they were infants, assuming Alexion sticks to a confidential per-patient cost cap and looks for further ways to cut down on the NHS’ short-term costs. But NICE wouldn’t recommend the product in children with juvenile-onset disease--a decision Alexion wasn’t happy with.

“We are disappointed that the draft guidance dismisses the needs of patients with juvenile-onset HPP and is in direct conflict with the EU approved label, which recommends Strensiq for all patients with paediatric-onset HPP,” the Connecticut company said in a statement.

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The way NICE sees it, Strensiq “could provide important clinical benefits” for some juvenile-onset patients. But the orphan drug also comes along with a lofty price tag--about £366,912 ($476,338) per patient for 52 weeks of treatment.

Alexion, as drugmakers often do, offered up a discount on the drug, putting forth a “managed access agreement” it developed in collaboration with physicians, the patient advocacy community and NHS England. But the end result was still more than the cost-effectiveness gatekeeper wanted to shell out--especially considering the “significant uncertainties” it sees regarding Strensiq’s long-term benefits and the treatable juvenile-onset patient population.

“By disregarding the consensus position developed directly with treating physicians and patient organizations, Alexion believes the NICE assessment process has fundamentally failed to appropriately evaluate the benefits of Strensiq for patients with pediatric-onset HPP,” the drugmaker’s statement said.

Still, NICE’s latest verdict is an improvement for Alexion, which is working to diversify beyond ultra-pricey orphan player Soliris. Back in December, NICE turned down Strensiq altogether, citing price constraints.

And Alexion recently found coverage success elsewhere, too, striking an agreement for Strensiq funding in Germany. Under that pact, the country will provide access for all patients with pediatric-onset HPP--including those that NICE wouldn’t cover, Alexion told FiercePharma in a statement.

- read NICE's guidance (PDF)

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Alexion's Soliris, Bayer's Xarelto get stiff-armed by a couple of cost gatekeepers
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