Novartis’ oral Fabhalta (iptacopan) has been approved by the US Food and Drug Administration (FDA) as the first treatment for the ultra-rare kidney disease complement 3 glomerulopathy (C3G).
Every year, approximately one to two people per million worldwide are diagnosed with C3G, in which deposits of C3 protein build up in the kidney glomeruli, the body’s network of blood vessels designed to filter waste and remove extra fluids from the blood.
This causes inflammation and glomerular damage that results in proteinuria (protein in urine), haematuria (blood in urine) and reduced kidney function, with about half of C3G patients progressing to kidney failure within ten years of diagnosis.
Fabhalta, which has been specifically authorised to reduce proteinuria in adults with C3G, selectively targets what is thought to be the underlying cause of the disease.
The drug is already approved in the US to treat patients with immunoglobulin A nephropathy, another rare kidney disease, and the rare blood disorder paroxysmal nocturnal haemoglobinuria.
The FDA’s latest decision was based on positive result from the phase 3 APPEAR-C3G study, which showed that patients being treated with Fabhalta alongside supportive care achieved a statistically significant and clinically meaningful 35.1% reduction in proteinuria at six months compared to those randomised to receive placebo.
Data on the secondary endpoint of estimated glomerular filtration rate, a measure of kidney function, also showed improvements over six months with Fabhalta compared to placebo, and Novartis’ therapy demonstrated a favourable safety profile with no new safety signals observed.
APPEAR-C3G study co-investigator, 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.
“This approval of Fabhalta is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients.”
The decision comes less than a month after Fabhalta was recommended by the European Medicines Agency’s human medicines committee to treat adults with C3G.
The European Commission will now review the Committee for Medicinal Products for Human Use’s recommendation as it makes a decision on Fabhalta in this indication.
