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After its $7.6B buyout fell through, Sobi scores NICE backing for rare disease drug Aspaveli

After rare disease drugmaker Sobi reportedly had a buyout scuppered by pharma giant AstraZeneca, the company has picked up a win that stands to bolster its Aspaveli launch.

England’s National Institute for Health and Care Excellence (NICE) has given a thumbs up to Sobi’s paroxysmal nocturnal hemoglobinuria (PNH) drug Aspaveli. NICE issued its decision after setting up Aspaveli for a speedy appraisal, Sobi said in a release.

NICE recommends Aspaveli as an option for adult PNH patients who are anemic after treatment with a C5 inhibitor, such as AstraZeneca’s Ultomiris, for at least three months. The recommendation assumes, of course, that Sobi’s drug will pass muster with U.K. drug regulators.

Aspaveli, which is already marketed in the U.S. by Apellis as Empaveli, is currently under review by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA).

NICE often rejects drugs on pricing concerns. Not so with Sobi’s Aspaveli, though the drug is likely to be pricey. In the U.S., Apellis’ Empaveli costs around $36,790 per 160-mL supply, according to Sobi licensed ex-U.S. rights to the medicine last year.

PNH is a rare, chronic blood disorder characterized by the destruction of healthy, oxygen-carrying red blood cells, Sobi said. The disease affects some 900 people in England. The disorder can lead to frequent blood transfusions and cause symptoms such as severe fatigue and anemia. If left untreated, the disease has a 10-year mortality rate of 29%.

Meanwhile, AstraZeneca’s Ultomiris snagged its NICE blessing in April after offering up a confidential discount. The verdict allows the drug to be used in patients with high disease activity who’ve remained stable on Soliris for at least six months. The drug is dosed by weight, but it typically costs around £50,000 every other month before the discount.

AstraZeneca picked up Ultomiris and Soliris in its $39 billion buyout of Alexion.

But the story of AZ and Sobi doesn’t end with Ultomiris and Aspaveli. AZ reportedly blocked an investor group’s proposed buyout of Sobi, multiple sources reported in early December. By holding back an 8% stake in Sobi, AstraZeneca effectively repelled an investor group’s $7.6 billion buyout of the rare disease specialist, Bloomberg reported, citing people close to the matter.