Novartis, a global pharmaceutical company, has received FDA accelerated approval for Fabhalta (iptacopan).
The therapeutic is the first and only complement inhibitor designed to reduce proteinuria in patients with primary IgA nephropathy (IgAN) — which is often called Berger's disease.
This results comes off the back of the Phase III APPLAUSE-IgAN interim analysis, which demonstrated Fabhalta's ability to reduce the prevalence of proteinuria in patients by 38% versus placebo, which was a clinically significant figure.
The decision marks Novartis' first approval from its renal development pipeline, which also includes atrasentan and zigakibart.
The continuation of this approval likely depends on Fabhalta's ability to offer a clinical benefit, while also being able to slow disease progression through the maintenance of glomerular filtration — which will be further explored in the APPLAUSE-IgAN trial.
What is Fabhalta?
Iptacopan is an inhibitor of the alternative complement pathway, which is a system believed to be involved in the pathogenesis of IgA nephropathies 1 to 4.
Despite the current standard of care available on the market, around 50% of IgA nephropathy patients with persistent proteinuria develop kidney failure within 10 to 20 years of diagnosis.
“The heterogeneous and progressive nature of IgA nephropathy has made it challenging to effectively treat this disease. Thankfully, the treatment landscape is rapidly evolving,” said Prof. Dana Rizk, Investigator and APPLAUSE-IgAN Steering Committee Member and professor in the University of Alabama at Birmingham Division of Nephrology. “Mounting clinical evidence highlights the pivotal role of complement activation in IgA nephropathy. I am thrilled that this advancement is now available to help enable a targeted treatment approach for IgAN patients.”
IgAN and its associated unmet needs
IgAN is a progressive, rare autoimmune disease that impacts the kidneys, often causing glomerular inflammation and proteinuria.
Around 25 people per million are diagnosed with IgAN each year, and disease progression can vary significantly between patients — as well as treatment responses.
Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients.
“Today’s approval of Fabhalta as a first-in-class medicine for IgA nephropathy is an important milestone in our journey to evolve rare renal disease care by bringing new treatments to people in urgent need of options,” said Victor Bultó, President US, Novartis. “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 broad portfolio.”