Novartis’ Fabhalta (iptacopan) has been granted accelerated approval by the US Food and Drug Administration (FDA) to reduce excess protein in the urine (proteinuria) of patients with the rare kidney disease immunoglobulin A nephropathy (IgAN).
Approximately 25 people per million worldwide are diagnosed each year with IgAN, which occurs when IgA accumulates in the kidneys and damages them. This impairs their filtering function and, as a result, the kidneys start to let substances such as blood and protein leak into the urine.
Despite current treatments, up to half of IgAN patients with persistent proteinuria progress to kidney failure within ten to 20 years of diagnosis.
Fabhalta is an oral factor B inhibitor of the alternative complement pathway and is already approved in the US to treat adults with the rare blood disorder paroxysmal nocturnal haemoglobinuria.
The FDA’s latest decision, which specifically applies to patients who are at risk of rapid disease progression, was supported by results from the ongoing APPLAUSE-IgAN study of twice-daily oral Fabhalta in adult IgAN patients on a stable dose of maximally-tolerated renin-angiotensin system inhibitor therapy with or without a stable dose of SGLT2i.
According to results from a pre-specified interim analysis of the phase 3 trial, patients treated with Fabhalta achieved a 38% proteinuria reduction at nine months compared to placebo.
The study also showed that Fabhalta was well tolerated, with a favourable safety profile that was consistent with previously reported data.
Victor Bultó, president US at Novartis, described the new approval as “an important milestone in [the company’s] journey to evolve rare renal disease care by bringing new treatments to people in urgent need of options”.
“We are deeply committed to those living with rare renal diseases and look forward to continued partnership with this community as we further advance our… portfolio,” Bultó said.
In line with the FDA’s accelerated approval pathway, the continued authorisation of Fabhalta may be contingent on the verification of clinical benefit from APPLAUSE-IgAN, which will continue to evaluate whether the therapy slows disease progression.
Beyond IgAN, Fabhalta is currently in development for a range of rare diseases, including C3 glomerulopathy, immune complex membranoproliferative glomerulonephritis, atypical haemolytic uraemic syndrome and lupus nephritis.
