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news.The US Food and Drug Administration (FDA) has granted approval for Novartis’ Fabhalta (iptacopan), intended to minimise proteinuria in the adult population with C3 glomerulopathy (C3G).
Usually diagnosed in the young adult population, C3G is stated to be a rare kidney condition. Credit: Robina Weermeijer on Unsplash.
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The therapy claims to be the only oral inhibitor targeting the alternative complement pathway, specifically addressing what is believed to be the root cause of the disease.
Usually diagnosed in the young adult population, C3G is stated to be a rare kidney condition that leads to its failure.
The two times-a-day Fabhalta’s safety and efficacy were assessed in the Phase III APPEAR-C3G trial for adult subjects.
The trial included a six-month double-blind, randomised treatment duration with the therapy versus placebo alongside supportive care.
An additional open-label treatment time of six months was followed, where all subjects received the therapy.
The therapy’s treatment led to proteinuria minimisation, which was observed as early as 14 days and continued at 12 months.
In the open-label duration, proteinuria minimisation was observed in subjects who transitioned to the therapy.
A “favourable” safety profile was shown by the therapy, without any safety signals observed.
In C3G subjects treated with the therapy, nasopharyngitis, and viral infections were observed as the common adverse events.
The therapy secured the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) positive opinion last month for treating C3G.
In Japan and China, regulatory reviews for C3G are underway.
Discovered by the company, Fabhalta gained initial approval in December 2023 from the US regulator for treating the adult population with paroxysmal nocturnal haemoglobinuria (PNH).
Novartis is investigating the therapy across a wide range of other rare kidney conditions, encompassing lupus nephritis (LN), atypical haemolytic uremic syndrome, and immune complex membranoproliferative glomerulonephritis.
Fabhalta APPEAR-C3G trial co-investigator and University of Iowa Pediatrics-Nephrology professor Carla Nester said: “C3G is a debilitating disease often affecting young people, impacting many aspects of their physical and emotional health, and our previous treatment options came with significant challenges.”
