FDA grants approval of pegcetacoplan (Empaveli) injection for treatment of paroxysmal nocturnal haemoglobinuria (PNH)

Approval based on RCT (n=80) which found pegcetacoplan superior to eculizumab with respect to change in haemoglobin level from baseline to week 16, with adjusted mean difference of 3.84g/dL (p<0.001).

Source:

FDA