FDA Grants Priority Review to Pegcetacoplan for C3G and IC-MPGN

The FDA has granted Priority Review to the C3/C3b inhibitor pegcetacoplan (Empaveli) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

Priority Review designation is granted to marketing applications for medicines that treat serious conditions, and if approved, provides a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition.

Pegcetacoplan is approved to treat adult patients with paroxysmal nocturnal hemoglobinuria. The supplemental New Drug Application (sNDA) for the C3G and IC-MPGN indications is supported by data from the phase 3 VALIANT study (NCT05067127).

VALIANT evaluated the efficacy and safety of pegcetacoplan in participants aged 12 years and older with native or post-transplant recurrent C3G or primary IC-MPGN. Participants were randomly assigned to receive pegcetacoplan subcutaneously or placebo twice weekly for 26 weeks. The primary end point was the log-transformed ratio of urine protein creatinine ratio (UPCR) at week 26 from baseline.

Treatment with pegcetacoplan was associated with a statistically significant 68% reduction in proteinuria compared with placebo (P <0.0001), and results were consistent across all subgroups. In addition, there were notable reductions in C3c staining (nominal P<0.0001) and clinically meaningful stabilization in estimated glomerular filtration rate in the pegcetacoplan group compared with the placebo group (nominal P=0.03).

The Prescription Drug User Fee Act target date for the pegcetacoplan sNDA is July 28, 2025.

Source: Press release