NICE Accepts ULTOMIRIS® (ravulizumab) for Use in Patients with Atypical Haemolytic Uraemic Syndrome (aHUS)

NICE Accepts ULTOMIRIS^® (ravulizumab) for Use in Patients with Atypical Haemolytic Uraemic Syndrome (aHUS)

Ravulizumab is the first approved long-acting C5 complement inhibitor for aHUS – a life-threatening, ultra-rare disease

UXBRIDGE – May 20, 2021 – Alexion Pharma UK has announced that the National Institute for Health and Care Excellence (NICE) has accepted ULTOMIRIS^® (ravulizumab) [https://www.nice.org.uk/guidance/gid-ta10564/documents/final-appraisal-determination-document] for use by the NHS in England for the treatment of patients with a body weight of 10kg or above with atypical haemolytic uraemic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received SOLIRIS^® (eculizumab) for at least 3 months and have evidence of response to eculizumab.[i] Ravulizumab is the first and only long-acting complement inhibitor that provides infusions every eight weeks (or every four weeks for patients 10-20kg). Ravulizumab provides immediate, complete, and sustained C5 inhibition, allowing the majority of patients to achieve a complete thrombotic microangiopathy (TMA) response and improvement in kidney function.

“This is great news for the aHUS community, as ravulizumab will reduce the number of infusions needed per year compared to eculizumab, significantly improving the quality of life for all those living with the condition,” said David Kavanagh, Professor of Complement Therapeutics at the National Renal Complement Therapeutics Centre (NRCTC). “Ravulizumab is a long-acting formula, thus increasing the time between infusions, providing similar efficacy with an acceptable safety profile. Having this choice is good news for all those living with aHUS and their families.”

The NICE approval of ravulizumab is based on a comprehensive clinical and health economic submission by Alexion, as well as evidence provided by patient advocacy organisations and clinical experts.

"The NICE decision is excellent news for aHUS patients and their families. Ravulizumab will make their lives much freer and easier whilst providing management of a life-threatening rare disease,” said Len Woodward, Director Trustee aHUS alliance Global Action.

“We are extremely pleased to see NICE also welcome ravulizumab for the treatment of patients with aHUS in England,” said Sean Richardson, Alexion General Manager, UK & Ireland. “We know how impactful these decisions are for the rare disease community, and each approval we receive advances the quality of life for patients. At Alexion, we are relentless in our ambition to innovate for patients and it brings great pleasure knowing that patients in England will benefit from the approval of this new therapeutic option.”

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About atypical haemolytic uremic syndrome (aHUS)

aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. aHUS occurs when the complement system—a part of the body’s immune system—over-responds, leading the body to attack its own healthy cells. aHUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis—in addition to treatment—is critical to improving patient outcomes. Available tests can help distinguish aHUS from other haemolytic diseases with similar symptoms.

About ULTOMIRIS^®

ULTOMIRIS^® (ravulizumab) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks, following a loading dose, to treat PNH in adults, and aHUS patients weighing >20kg (every four weeks for aHUS patients weighing 10-20kg). ULTOMIRIS is approved in the UK for the treatment of adults and children with a body weight of at least 10kg with atypical haemolytic uremic syndrome (aHUS). ULTOMIRIS is also approved in the UK for treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) in patients with haemolysis with clinical symptom(s) indicative of high disease activity and in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development, and commercialisation of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed two approved complement inhibitors to treat patients with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of haematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology, and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Further information about Alexion can be found at: www.alexion.com/worldwide/UK.

[i] NICE. Final appraisal document. Ravulizumab for treating atypical haemolytic uraemic syndrome. Available at: https://www.nice.org.uk/guidance/gid-ta10564/documents/final-appraisal-determination-document. Last accessed: May 2021