The US Food and Drug Administration (FDA) has granted approval to Apellis Pharmaceuticals along with partner Sobi for Empaveli (pegcetacoplan) to address two uncommon kidney conditions. Empaveli, a C3-targeted therapy, is now authorized for reducing excessive protein in the urine in patients aged 12 and above diagnosed with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
These two conditions, affecting an estimated 5,000 individuals in the US, are characterized by an accumulation of the C3 protein, a crucial indicator of disease activity that can lead to kidney inflammation, damage, and failure. Notably, about 50% of patients face kidney failure within five to ten years of diagnosis.
Empaveli, designed to regulate the overactivation of the complement cascade in the immune system, has already been approved in the US for treating paroxysmal nocturnal hemoglobinuria, a rare blood disorder. The FDA’s decision was backed by positive outcomes from the VALIANT trial, where Empaveli showcased a 68% reduction in proteinuria compared to a placebo.
Additionally, Empaveli demonstrated kidney function stabilization, measured by the estimated glomerular filtration rate, and effective clearance of C3 deposits, as indicated by C3 staining. These benefits were consistent across both adolescent and adult patients with C3G and primary IC-MPGN, including those with post-transplant disease recurrence.
Apellis Pharmaceuticals’ co-founder and CEO, Cedric Francois, highlighted the transformative potential of Empaveli for patients with C3G and primary IC-MPGN, emphasizing the scarcity of treatment options available until now. The drug’s ability to control the three key disease markers in the largest pivotal study of these conditions signifies a significant advancement in patient care.
Apellis holds exclusive commercialization rights for systemic pegcetacoplan in the US, with Sobi having these rights elsewhere. The company also possesses global commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. Lead principal investigator of the VALIANT study, Carla Nester, emphasized the critical impact of Empaveli’s approval in addressing the urgent treatment needs of patients with these severe and rare kidney diseases.
The approval of Empaveli by the FDA marks a pivotal moment in the treatment landscape for rare kidney diseases, offering new hope to patients facing the challenges of C3G and primary IC-MPGN. The positive outcomes from the VALIANT trial highlight Empaveli’s potential to not only reduce proteinuria but also stabilize kidney function and clear C3 deposits, presenting a promising therapeutic option for patients with these conditions. The exclusive commercialization rights held by Apellis in the US and its global presence in the ophthalmological field position the company for continued growth and impact in the rare disease treatment space.
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