Iptacopan is recommended, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia. Iptacopan is only recommended if the company provides it according to the commercial arrangement.
Why the committee made this recommendation
Standard care for PNH with haemolytic anaemia includes eculizumab and ravulizumab, which are C5 inhibitors. Most people with PNH have ravulizumab. People who still have anaemia after having a C5 inhibitor usually have pegcetacoplan or ravulizumab.
Evidence from clinical trials shows that iptacopan increases the level of haemoglobin in the blood and reduces the need for blood transfusions.
For people who have not had a C5 inhibitor, an indirect comparison suggests that iptacopan is more effective than eculizumab and ravulizumab, but these results are uncertain.
For people who still have anaemia after having a C5 inhibitor, clinical trial evidence shows that iptacopan is more effective than eculizumab and ravulizumab. An indirect treatment comparison suggests that iptacopan is more effective than pegcetacoplan, but the results are uncertain.
The cost-effectiveness estimates for iptacopan are within the range that NICE considers an acceptable use of NHS resources. So, it is recommended.