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NICE recommends ULTOMIRIS ® (ravulizumab) for patients with Paroxysmal Nocturnal H a emoglobinuria (PNH)

NICE recommends ULTOMIRIS ® (ravulizumab) for patients with Paroxysmal Nocturnal H a emoglobinuria (PNH)

– Ravulizumab is the first a pproved , long acting C5 complement inhibitor for PNH administered every other month

UXBRIDGE APRIL 15th, 2021 – Alexion Pharma UK has announced that tthe National Institute for Health and Care Excellence (NICE has recommended ULTOMIRIS ® ( for use in the

National Health Service ( NHS for the treatment of paroxysmal nocturnal h a emoglobinuria (PNH) in adult patients with haemolysis with clinical symptoms suggesting high disease activity, or whose disease is clinically stable after having SOLIRIS ® (eculizumab) for at least 6 1 As the first and only long acting C5 complement inhibitor r avulizumab represents a step forward in the treatment of PNH, delivering comparable safety and efficacy to eculizumab with infusions every eight weeks compared to every two weeks.

"The availability of ravulizumab will have a meaningful impact on the lives of patients with PNH,” said Professor Peter Hillmen, Professor of Experimental Haematology at the University of Leeds and Honorary Consultant in Clinical Haematology at Leeds Teaching Hospitals NHS Trust. “Ravulizumab is as effective as the previous standard of care, eculizumab, with a similar safety profile, but with a longer-acting formulation. Patients with PNH will now only require infusions every 8 weeks rather than every 2 weeks which will have a major impact on their quality of life.”

PNH is a debilitating ultra rare blood disorder characteri s ed by complement mediated destruction of red blood cells (haemolysis) as well as risk of blood clots ( thrombosis), which can occur throughout the body, and result in organ damage and premature death 2 It impacts approximately 16 people per million in the United Kingdom 3 It can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s 4 5 PNH often goes unrecognised, with delays in diagnosis ranging from one to more than five years.6 The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis in addition to appropriate treatment is critical to improving patient outcomes.

“We are delighted at the recommendation from NICE, meaning we will be able to offer ravulizumab as a new treatment option to adult patients with PNH across the UK, helping to improve their overall quality of life,” said Sean Richardson, Alexion General Manager, UK & Ireland. “We believe ravulizumab will become the new standard of care for patients with PNH by providing immediate and complete C5 inhibition, sustained throughout the eight-week dosing interval.”

The NICE recommendation of ravulizumab is based on the assessment of a comprehensive clinical and health economic submission by Alexion, as well as evidence provided by patient advocacy organi s ations and clinical experts.

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Last Updated: 15-Apr-2021