Pegcetacoplan for treating paroxysmal nocturnal haemoglobinuria

1.1 Pegcetacoplan is recommended, within its marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults who have anaemia after at least 3 months of treatment with a C5 inhibitor. It is recommended only if the company provides pegcetacoplan according to the commercial arrangement.

Why the committee made these recommendations

Current treatments for PNH include C5 inhibitors such as eculizumab and ravulizumab. Some people still experience anaemia and symptoms of PNH while having these treatments. Clinical trial evidence suggests that pegcetacoplan improves haemoglobin levels (a measure of anaemia) and haematological symptoms of PNH for people who have anaemia while taking eculizumab. Pegcetacoplan is likely to have the same clinical benefits for people who have anaemia while taking ravulizumab, because ravulizumab is very similar to eculizumab.

For adults with anaemia while having a C5 inhibitor, pegcetacoplan is more effective and costs less than ravulizumab and eculizumab. Therefore, it is recommended.