Apellis Scores Double Approval for Empaveli in Rare Kidney Diseases, Challenging Novartis

Apellis Pharmaceuticals has secured a significant victory in the rare kidney disease market with a dual FDA approval for its complement inhibitor Empaveli (pegcetacoplan). The drug has been cleared to treat both complement 3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in adults and adolescents, marking a notable advancement in the treatment of these severe conditions.

Empaveli's Broad Label Outmaneuvers Novartis

Apellis's Empaveli has emerged as a formidable competitor to Novartis' Fabhalta in the C3G space, boasting a broader label that includes both adult and adolescent patients. Unlike Fabhalta, which is limited to adult C3G patients, Empaveli's approval extends to the IC-MPGN market, where it stands as the first FDA-approved treatment. This expanded scope positions Apellis to potentially capture a larger share of the rare kidney disease market.

The FDA's decision was based on the results of the phase 3 VALIANT study, which demonstrated Empaveli's efficacy across a wide range of patients in both C3G and IC-MPGN. The trial showcased impressive outcomes in key kidney disease measures, including a 68% reduction in proteinuria at week 26 compared to placebo. Additionally, the drug showed consistent results across age groups and among patients with native or post-transplant kidney status in both diseases.

Clinical Efficacy and Market Potential

Empaveli's performance in the VALIANT study has been described as showing "enormous efficacy" by analysts at Evercore ISI, who view the drug as a "blockbuster opportunity." The drug's mechanism of action, which regulates excessive activation of the immune system's complement cascade, is central to its effectiveness in treating these rare kidney diseases.

Key clinical findings include:

• 68% reduction in proteinuria compared to placebo at week 26
• Substantial proportion of patients showing reduction in C3c staining intensity
• 71% of treated patients achieving complete clearance of C3c staining over placebo

These results are particularly significant given the severity of C3G and primary IC-MPGN, which can lead to kidney failure in about half of patients within five to ten years of diagnosis. Approximately 90% of patients who receive a kidney transplant experience disease recurrence, underscoring the critical need for effective treatments.

Strategic Positioning and Market Readiness

Apellis CEO Cedric Francois emphasized the company's preparedness to address the "incredible need" in this disease area. The company has expanded its team to include kidney care experts and is leveraging its existing infrastructure from its paroxysmal nocturnal hemoglobinuria (PNH) indication, with around 70% of U.S. patients with C3G and IC-MPGN being treated in the same care centers.

With this approval, Apellis continues its growth trajectory, having secured three approvals in four years. The company's portfolio now includes Empaveli for PNH and rare kidney diseases, as well as Syfovre, an injectable version of pegcetacoplan for geographic atrophy. Francois considers both drugs to have potential blockbuster status, with Citi Research analysts predicting that the new approvals will "transform" the Empaveli franchise.

As Apellis prepares to make what Francois calls an "enormous difference" in patients' lives, the pharmaceutical landscape for rare kidney diseases appears set for increased competition and potentially improved patient outcomes.