FDA Priority Review for Empaveli

The sNDA submission is supported by positive Phase III VALIANT results at Week 26

The positive results were consistent across patients with C3G and IC-MPGN, adolescents and adults, and native and post-transplant kidney disease.

• 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 I-treated patients showed complete clearance of C3c staining compared to placebo.
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   Empaveli showed favorable safety and tolerability, consistent with its established profile.
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• The VALIANT Phase III study (NCT05067127) is a randomized, placebo-controlled, double-blinded, multi-center study designed to evaluate pegcetacoplan efficacy and safety in 124 patients who are 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include adolescent and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients are able to proceed to a 26-week open-label phase in which all patients receive pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.
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• "EMPAVELI demonstrated clinically meaningful benefits across all three key markers of disease – unprecedented proteinuria reductions, stabilization of kidney function, and substantial clearance of C3c staining,” said Dr. Bradley P. Dixon, professor of pediatrics and medicine, University of Colorado School of Medicine, and co-investigator of the VALIANT study. “Efficacy is critically important when treating C3G and IC-MPGN given the high risk of progression to kidney failure. As a disease-modifying therapy, EMPAVELI has the potential to make a life-changing difference for patients, if approved.”

“This milestone represents a significant step toward our goal of bringing EMPAVELI to people living with C3G and primary IC-MPGN, regardless of their disease type, age, or transplant status,” said Dr. Cedric Francois, chief executive officer and co-founder at Apellis. “We look forward to working with the FDA to make this treatment available to patients in need as quickly as possible.”