Apellis Pharmaceuticals Secures FDA Approval for Empaveli in Rare Kidney Disorders

Apellis Pharmaceuticals has secured FDA approval for Empaveli to treat two rare kidney disorders, expanding its previous indication for paroxysmal nocturnal hemoglobinuria. Empaveli is a C3 inhibitor that will now be available for patients with membranoproliferative glomerulonephritis and IgA nephropathy. The approval is a significant milestone for Apellis, allowing the medication to reach more patients with rare kidney disorders.

Apellis Pharmaceuticals has secured FDA approval for Empaveli (pegcetacoplan) to treat two rare kidney disorders, expanding its previous indication for paroxysmal nocturnal hemoglobinuria. Empaveli is a C3 inhibitor that will now be available for patients with membranoproliferative glomerulonephritis (IC-MPGN) and IgA nephropathy. This approval marks a significant milestone for Apellis, allowing the medication to reach more patients with rare kidney disorders.

Empaveli demonstrated its effectiveness in a Phase 3 clinical trial, known as VALIANT, which showed a 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits compared to placebo. These positive results were consistent across adolescent and adult patients with C3 glomerulopathy (C3G) and primary IC-MPGN, as well as in C3G patients with post-transplant disease recurrence [1].

The FDA's approval of Empaveli for these rare kidney disorders is based on the positive six-month results from the VALIANT study. The study demonstrated significant benefits across three key markers of disease: proteinuria reduction, stabilization of kidney function, and reduction of C3 staining intensity. Empaveli-treated patients achieved a 68% reduction in proteinuria, stabilization of kidney function, and a majority achieved a reduction in C3 staining intensity, with 71% achieving complete clearance of C3 deposits [1].

Empaveli's safety profile is well-established, with over 2,200 patient years of exposure across all approved indications. The most common adverse reactions in the VALIANT study were infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea. The drug will be marketed with a Boxed Warning and distributed under an FDA safety program called Risk Evaluation and Mitigation Strategy (REMS) due to its potential to increase the risk of serious infections [2].

With this new approval, Apellis Pharmaceuticals now has three FDA approvals in four years, demonstrating its commitment to serving the "incredible need" in the rare kidney disease area. Empaveli's potential to preserve kidney function by controlling all three key markers of disease positions it as a "blockbuster opportunity," according to analysts [3].

References:

[1] https://www.americanpharmaceuticalreview.com/1315-News/620570-FDA-Approves-EMPAVELI-for-Kidney-Disease/
[2] https://seekingalpha.com/news/4473655-apellis-wins-fda-label-expansion-empaveli
[3] https://www.fiercepharma.com/pharma/apellis-counters-novartis-broad-label-empaveli-kidney-disease