Novartis and Apellis Pharmaceuticals are competing to develop treatments for a rare kidney disease. Both companies recently shared promising results from their research on new drugs.
Apellis presented detailed results from a late-stage trial of their drug, pegcetacoplan, which is aimed at treating kidney diseases called C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). The drug showed success in reducing protein in the urine, a key indicator of kidney disease, by over 68% within 24 weeks. It also helped stabilize kidney function and reduced disease markers in patients.
Pegcetacoplan is already approved for other conditions under the names Empaveli and Syfovre. Apellis plans to seek U.S. approval for this new use in early 2025.
Novartis also reported positive results for their drug, iptacopan, marketed as Fabhalta. It showed significant reductions in urine protein levels over a year and improved kidney function in patients with C3G. Novartis has already submitted applications for approval in Europe, China, and Japan and plans to file in the U.S. soon.
Both drugs were generally safe in the trials. Currently, there are no treatments targeting the root causes of C3G and IC-MPGN, which often lead to kidney failure. Apellis estimates these diseases affect about 5,000 people in the U.S. and up to 8,000 in Europe.
Experts believe pegcetacoplan has strong potential to dominate the market due to its effectiveness, making it a potential blockbuster in the treatment of these rare kidney diseases.