FDA Reviews Kidney Disease Drug From Apellis Pharmaceuticals

The U.S. Food and Drug Administration (FDA) on Tuesday accepted and granted Priority Review designation to Apellis Pharmaceuticals, Inc.’s (NASDAQ:APLS) supplemental New Drug Application (sNDA) for Empaveli (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

C3G and primary IC-MPGN are rare kidney diseases. Excessive C3c deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.

Also Read: JPMorgan Cut Apellis’ Price Forecasts, Cites Longer Peak Sales Timing For Syfovre

The Prescription Drug User Fee Act (PDUFA) target action date is July 28, 2025.

The supplemental application submission supported by Phase 3 VALIANT results at Week 26:

• Proteinuria reduction: The study met its primary endpoint, demonstrating a statistically significant 68% (p<0.0001) proteinuria reduction in Empaveli-treated patients compared to placebo.
• Stabilization of kidney function: Empaveli-treated patients achieved stabilization of kidney function (nominal p=0.03) as measured by eGFR.
• Reduction of C3c staining: A substantial proportion of Empaveli-treated patients achieved a reduction in C3c staining intensity (nominal p<0.0001). 71% of Empaveli-treated patients showed complete clearance of C3c staining compared to placebo.

Empaveli showed favorable safety and tolerability, which is consistent with its established profile.

William Blair writes, “We believe the recent approval of Fabhalta (iptacopan) for the treatment of adults with C3G bodes well for an approval of Empaveli given it demonstrated what we believe is best-in-class data and did so in a broader population that included adolescents, patients with IC-MPGN, and post-transplant patients, which should support a broader label and larger addressable market.”

In March, the FDA approved Novartis AG’s (NYSE:NVS) oral Fabhalta (iptacopan) for adults with C3 glomerulopathy (C3G) to reduce proteinuria (protein in the urine), making it the first and only treatment approved for this condition.

Price Action: APLS stock is up 8.05% at $23.63 at the last check Tuesday.

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