Conference report: Pegcetacoplan (Empaveli) demonstrated sustained normalisation of clinical measures in treatment-naïve patients with paroxysmal nocturnal haemoglobinuria in Phase 3 PRINCE study
In RCT (n=53), treatment resulted in superior improvements in co-primary endpoints of haemoglobin stabilisation through week 26 (46% vs. 0%, p<0.0010) and reduction in lactate dehydrogenase, compared to standard of care, which did not include complement inhibitors, at week 26.
Source:
Biospace Inc.
SPS commentary:
Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade. The European CHMP issued a positive opinion in October supporting approval for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). In DRAFT guidance, NICE recommended pegcetacoplan as an option for treating PNH in adults who have anaemia after at least 3 months of treatment with a C5 inhibitor.