Novartis’ Fabhalta (iptacopan) has been recommended by the European Medicines Agency’s human medicines committee to treat adults with the ultra-rare kidney disease complement 3 glomerulopathy (C3G).
Approximately one to two people per million globally are diagnosed every year with C3G, which occurs when an over-activation of the alternative complement pathway causes deposits of C3 protein to build up in kidney glomeruli.
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.
There are currently no approved treatments for C3G and, if approved by the EC, Fabhalta would be the only medicine indicated to selectively target the underlying cause of the disease.
Novartis’ Fabhalta is an oral, factor B inhibitor of the alternative complement pathway and is already approved in the EU to treat adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria.
The CHMP’s latest recommendation of the drug was supported by 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 steering committee member, David Kavanagh, the National Renal Complement Therapeutics Centre at Newcastle University, said: “C3G has no approved treatments, and patients face challenges with current options.
“With its strong body of evidence, oral Fabhalta targets the underlying cause of C3G in both native and recurrent patients and can bring hope to patients who currently have a poor prognosis.”
David Soergel, global head, cardiovascular, renal and metabolism development unit at Novartis, added that the positive CHMP opinion “marks an important step forward for [Novartis’] exciting multi-asset kidney pipeline, underscoring [the company’s] commitment to making meaningful progress for patients with unmet needs”.
The European Commission will now review the CHMP’s recommendation as it makes a decision on Fabhalta in this indication, expected within two months.
