Alexion and the UK’s National Institute for Health and Care Excellence (NICE) have agreed a confidence commercial discount for the paroxysmal nocturnal haemoglobinuria (PNH) treatment Ultomiris for use within the NHS.
The cost-effectiveness watchdog has backed the use of Ultomiris (ravulizumab) to treat PNH in adults with haemolysis with clinical symptoms suggesting high disease activity, or whose disease is clinically stable after receiving Alexion’s older PNH drug Soliris (eculizumab) for six months or more.
PNH is a rare blood condition, wherein red blood cells are destroyed which can result in blood clots and impaired bone marrow function.
According to Johns Hopkins Medicine, the condition affects between 1-1.5 million people per million of the population, with the median age of diagnosis being 34-50 years old.
In its final appraisal document, NICE said clinical evidence shows Ultomiris is as safe and effective as Soliris and also is administered less often, meaning there is some benefit on quality of life. The less frequent dosing of Ultomiris may also save costs, NICE added.
The list price for Ultomiris in the UK is £4,533 per 300 mg/3 ml concentrate and £16,621 per 1,100 mg/11 ml concentrate.
NICE determined that Ultomiris was more effective and less costly than Soliris ‘in all scenario analyses presented’, including the confidential discount for the drug.
Compared with Alexion’s older drug, Ultomiris is a cost-effective use of NHS resources, concluded NICE.
Alexion’s follow-up PNH therapy Ultomiris was initially granted a US Food and Drug Administration (FDA) approval in December 2018.
In clinical trials, Ultomiris matched Soliris for efficacy, offering improved dosing – it is administered intravenously every eight weeks rather than every two weeks, slashing the number of doses required from 26 to six per year.
Alexion is aiming to grow sales of Ultomiris ahead of expected biosimilar competition on Soliris in key markets.
In 2020, Alexion reached a patent settlement with Amgen, preventing a biosimilar version of the drug from entering the US marketing until 2025.
However, in the EU, Alexion’s attempt to extend patent protection for Soliris fell flat after the European Patent Office rejected the company’s efforts to prolong two patents for the drug.
As a result, Soliris biosimilars are expected to enter the EU market as early as 2022.