Alexion's pricey Ultomiris wins backing from England's cost watchdogs thanks to confidential discount

Alexion has added a nice piece to Ultomiris’ growth potential ahead of its planned merger with AstraZeneca, scoring backing from England’s drug cost watchdog in a key indication.

The C5 inhibitor now has full coverage on the National Health Service for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), thanks to a recommendation from the National Institute for Health and Care Excellence (NICE). It can now be used in patients with high disease activity who have remained stable on the drug’s predecessor Soliris for at least six months.

Alexion earned Ultomiris’ support from NICE after offering up a confidential discount. The drug’s list price in the U.K. is £4,533 per 300 mg/3 ml concentrate, and £16,621 for the 1,100 mg/11 ml concentrate.

The drug is dosed by weight. In the 60 kg to 100 kg weight range, the maintenance dose is 3,300 mg, or three 1,100-mg vials, every eight weeks. That means it costs around £50,000 every other month before the discount.

NICE’s reviewers view Ultomiris as similarly effective and just as safe as Alexion’s own Soliris, which is dosed every other week. Ultomiris’ longer interval offers patients quality of life benefits and may also save costs, NICE’s appraisal experts noted.

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Bracing for a potential biosimilar entry in 2025, Alexion has been focusing on converting Soliris patients to Ultomiris. As of the end of last year, the company had turned over 70% PNH patients to Ultomiris in major markets. It aims to achieve the same in atypical hemolytic uremic syndrome (HUS) within two years of launch, or at the end of this year in the U.S.

Thanks to the quick conversion, Ultomiris sales surged pass the blockbuster threshold last year, reaching $1.08 billion versus $339 million in 2019. Soliris also registered a small growth of 3% to $4.06 billion.

Overall, Alexion’s sales haul came in at $6.07 billion, up from $4.99 billion a year ago. The company is targeting annual global sales of $9 billion to $10 billion by 2025.

NICE is also considering Ultomiris coverage in atypical HUS, having held the first appraisal committee meeting for that use on Tuesday.

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Besides PNH and HUS, Alexion is simultaneously testing Ultomiris in phase 3 trials of generalized myasthenia gravis, neuromyelitis optica spectrum disorder and amyotrophic lateral sclerosis.

Boasting two blockbuster drugs and expertise in complement biology and more broadly, immunology, Alexion recently attracted AstraZeneca in a $39 billion buyout offer. AZ believes the complement cascade’s role in the innate immune system could mean broader applications of drugs targeting it beyond those rare diseases. Potential therapeutic fields AZ is eying with the buyout include oncology, neurology and respiratory, among others.