Danicopan is recommended, as an add-on to ravulizumab or eculizumab as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults who have residual haemolytic anaemia, only if:
they have clinically significant extravascular haemolysis while on treatment with a complement component 5 inhibitor (C5 inhibitor) and
the company provides it according to the commercial arrangement.
This recommendation is not intended to affect treatment with danicopan that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop.
Why the committee made these recommendations
Standard care for PNH with haemolytic anaemia includes the C5 inhibitors ravulizumab and eculizumab. After a C5 inhibitor, people who still have anaemia (residual haemolytic anaemia) and symptoms of PNH usually have pegcetacoplan. For this evaluation, the company asked for danicopan as an add-on to ravulizumab or eculizumab to be considered only for PNH in adults who have clinically significant extravascular haemolysis. This does not include everyone who it is licensed for.
Evidence from clinical trials shows that danicopan with a C5 inhibitor increases haemoglobin levels and reduces the need for blood transfusions more than a C5 inhibitor alone. There is no direct evidence comparing danicopan with pegcetacoplan and the results from an indirect comparison are uncertain.
The economic evidence for danicopan add-on treatment has some uncertainties, including long-term breakthrough haemolysis rates and around some of the assumptions used to estimate cost effectiveness. But, the most likely cost-effectiveness estimates are within the range usually considered a cost-effective use of NHS resources. So, danicopan is recommended.