The U.S. Food and Drug Administration (FDA) has granted approval to Empaveli (pegcetacoplan) as the primary treatment for C3 glomerulopathy (C3G).
The drug has received approval for patients over 12 years and above who work to minimise proteinuria with these rare disorders that affect the nephrons in the kidney.
Among Empaveli-treated patients, 71 percent achieved zero C3 staining intensity, showing complete clearance of C3 deposits. The results were consistent across adolescent and adult patients for both C3G and primary IC-MPGN, as well as in C3G patients with posttransplant disease recurrence.
The approval is supported by the phase 3 VALIANT study, in which patients consuming Empaveli noticed a 68% reduction in proteinuria, and a significant clearance of C3 deposits than placebo.
Among the patients who received the treatment for Empaveli, up to 71% achieved zero C3 staining intensity, showing clearance of C3 deposits.
It showed consistent progress across adult patients for both primary IC-MPGN and C3G, and in C3G patients with posttransplant disease recurrence.
However, the adverse events include fever, nasal congestions, throat, cough, nausea, and influenza.
Carla Nester, M.D., lead principal investigator for the VALIANT study from the University of Iowa in Iowa City, released a statement, which reads, "With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant."
"Given the urgent need, particularly in children, the approval of Empaveli marks a pivotal moment in the treatment of rare kidney diseases."
Apellis Pharmaceuticals received approval for Empaveli.
