Ultomiris Available Soon for Eligible aHUS Patients in England

Alexion Pharmaceuticals’ long-acting Ultomiris (ravulizumab) was added to the list of medications available through England’s national health service (NHS) to adults and children weighing at least 10 kilograms (22 pounds), with atypical hemolytic uremic syndrome (aHUS).

Ultomiris is Alexion’s successor to Soliris (eculizumab) — also approved for aHUS and given every other week — and allows for an eight-week dosing schedule after a loading phase.

While both therapies block the activity of the complement C5 protein, preventing the overactivation of the complement system and red blood cells’ destruction, which characterize aHUS, Ultomiris remains active for longer in the body, requiring less-frequent into-the-vein administrations. Of note, the complement system comprises a set of more than 30 blood proteins that form part of the body’s immune system.

As such, Alexion’s next-generation, long-acting C5 inhibitor has the potential to lower the burden and overall healthcare costs for patients, while improving their quality of life.

The recommendation was made by the National Institute for Health and Care Excellence (NICE), which advises the NHS on which treatments should be available. It covers patients who have never received a complement inhibitor, and those currently responding to Soliris, given for at least three months.

Ultomiris is expected to be available to the estimated 160 eligible aHUS patients in England within the next three months.

“Living with a rare blood condition can be both physically and mentally challenging, especially when frequent treatment is required,” Meindert Boysen, deputy chief executive and director of NICE’s Centre for Health Technology Evaluation, said in a press release.

“We are hopeful that the increased time between doses with this new treatment will lead to a better quality of life for these individuals and their loved ones,” Boysen added.

The recommendation follows a similar, recent decision in Scotland, and comes nearly a year after the therapy was approved by the European Commission for the same indication. NICE recommendations usually are followed by health authorities in Northern Ireland and Wales, where recommendations usually are implemented within two months following NICE’s appraisal.

The therapy also is approved in the U.S. and Japan for similar indications.

“Both the NICE’s and [Scottish Medicines Consortium’s] decisions will be  excellent news for patients in the UK,” as Ultomiris “will make their lives freer and easier while keeping them safe from aHUS, a harmful, life threatening and very rare disease,” the aHUS alliance Global Action stated in a separate press release.

“We hope that this will be the case in other countries around the world too,” added Global Action, an umbrella group of aHUS advocates and patient organizations in more than 30 countries.

By providing the opinion and experience of aHUS patients about the use of Ultomiris and the potential impact of the first and only long-lasting C5 inhibitor on patients and carers, the alliance also contributed to NICE’s decision.

NICE took into account data from Alexion providing evidence of Ultomiris’ favorable cost-effectiveness profile, as well as advice from aHUS clinical experts about the new therapy’s benefits, both relative to currently available treatments in the country.

Clinical evidence of Ultomiris’ favorable safety and effectiveness profiles was derived mainly from two ongoing Phase 3 clinical trials: aHUS-311 (NCT02949128), enrolling patients ages 12 and older; and aHUS-312 (NCT03131219), enrolling patients up to 17 years old. Most participants in both studies had not been treated with a complement inhibitor.

The therapy was found to safely and effectively resolve aHUS acute and life-threatening complications, resulting in stable kidney and blood measurements. Also, data from 10 children who participated in aHUS-312 and were on Soliris previously showed that switching to Ultomiris kept their disease under control without causing new safety concerns.

While Ultomiris has not been compared directly with Soliris in clinical trials, results of indirect comparisons suggest that the next-generation C5 inhibitor is as effective for treating aHUS, mainly due to its similar mechanism of action, NICE noted in its final appraisal document.

In addition, when equivalent efficacy was assumed, Ultomiris was less costly than Soliris and showed a cost-effectiveness profile within what NICE normally considers an effective use of NHS resources.

NICE’s recommendation on Ultomiris, particularly its statements on the therapy’s cost-effectiveness, will be considered for review when biosimilars of Soliris become available, which is expected after the therapy’s patent expires in the next three years. Similar to generics, biosimilars are low-cost alternatives to brand medications that are as safe and effective as the original products upon which they are based.

Ultomiris also was recommended for adults with paroxysmal nocturnal hemoglobinuria, a serious blood disorder caused by complement dysfunction. The therapy already is approved for this indication in the EU, U.K., U.S., and Japan.

Blake Dark, NHS England’s commercial medicines director, said that “hundreds of NHS patients will have access to [Ultomiris], a potentially life-improving treatment that could give patients with rare blood disorders a greater quality of life, more independence with fewer hospital admissions and blood transfusions throughout their lifetime.”

“The NHS is here for everyone, whether it’s providing COVID-19 vaccines for millions or securing access to innovative new medicines, and adopting new treatments such as [Ultomiris] demonstrates how the NHS can improve the lives of people suffering with rare chronic conditions and also secure the best value for the taxpayer,” Dark added.