FDA Approves EMPAVELI as the First Treatment for C3G or Primary IC-MPGN

The U.S. Food and Drug Administration (FDA) has approved Apellis Pharmaceuticals’ EMPAVELI (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States.

Approval was based on the Phase III VALIANT study that demonstrated an unprecedented 68 percent reduction in proteinuria, stabilization of kidney function and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence.

The safety profile of EMPAVELI is well-established, with more than 2,200 patient years across all approved indications. The most common adverse reactions in the VALIANT study (greater than or equal to 10 percent) were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough and nausea.

“EMPAVELI has the potential to be truly transformational for patients with C3G and primary IC-MPGN, who until now have had very few treatment options. In the largest pivotal study of these diseases, EMPAVELI demonstrated its potential to preserve kidney function by controlling all three key markers of disease,” said Cedric Francois, MD, PhD, co-founder and chief executive officer of Apellis. “As Apellis’ third approval in four years, this milestone underscores the unique ability of targeting C3 to improve patients’ lives. We are deeply grateful to everyone who made this approval possible and look forward to building on this momentum as we advance pivotal studies of EMPAVELI in other rare kidney diseases.”