Pegcetacoplan

Does Pegcetacoplan work?

Yes, in controlled trials, though small because the diseases are rare; one had 80 patients.

The PNH approval rests on PEGASUS, a randomized open-label phase 3 trial in 80 patients comparing pegcetacoplan with eculizumab, which showed a large hemoglobin advantage at 16 weeks. The kidney approvals rest on the phase 3 VALIANT trial. The trials are small because the diseases are rare; the effects are large and on hard laboratory endpoints. ¹³

PEGASUS enrolled 80 adults with PNH who stayed anemic despite at least three months of eculizumab. For four weeks they took both drugs. Then they were randomized. Some got pegcetacoplan alone. Others kept taking eculizumab. This lasted 16 weeks. The primary outcome was change in hemoglobin. It favored pegcetacoplan by about 3.8 grams per deciliter. Most patients on pegcetacoplan no longer needed transfusions. A minority on eculizumab did not need them. Signs of red-cell destruction improved. So did fatigue scores. ¹

For the kidney approved uses, the phase 3 VALIANT trial enrolled patients 12 and older. They had C3 glomerulopathy or primary IC-MPGN. Urine protein-to-creatinine ratio fell by about 68 percent relative to placebo. Kidney function held steady, and the FDA cited this in the July 2025 approval. ³

Read the full Pegcetacoplan page

Reference information only, not medical advice. Drawn from the Pegcetacoplan article, fact-checked against the cited sources. No dosing anywhere on this site.