Pegcetacoplan (Aspaveli®) approved in EU as orphan drug for treatment of paroxysmal nocturnal haemoglobinuria (PNH)
This is 1st targeted C3 therapy approved in EU for treatment of adults with PNH who are anaemic after treatment with a C5 inhibitor for ≥3 months. Approval was based on PEGASUS phase 3 study where it demonstrated superiority to eculizumab in improving haemoglobin levels.
Source:
Biospace Inc.
SPS commentary:
In recent DRAFT guidance, NICE recommended pegcetacoplan as an option for treating paroxysmal nocturnal haemoglobinuria in adults who have anaemia after at least 3 months of treatment with a C5 inhibitor.