Novartis’ Fabhalta receives FDA approval for treatment of rare kidney disorder

SWITZERLAND—Swiss pharmaceutical giant Novartis has received accelerated approval from the U.S. Food and Drug Administration (FDA) for Fabhalta (iptacopan), a drug used to treat rare kidney diseases.

This innovative drug is designed to reduce proteinuria in adults suffering from primary IgA nephropathy (IgAN), a rare and progressive kidney disorder.

 Fabhalta works by specifically targeting the immune system’s alternative complement pathway, serving as an inhibitor of this pathway.

The FDA’s approval of Fabhalta is grounded in the pre-specified interim results from the Phase III APPLAUSE-IgAN study, a pivotal trial that demonstrated the drug’s effectiveness.

The study showed that after nine months, Fabhalta achieved a remarkable 44% reduction in proteinuria compared to a placebo.

The APPLAUSE-IgAN study is a large-scale, multicenter, randomized, double-blind, placebo-controlled trial involving 518 adult patients with IgAN.

The primary goals of the trial include evaluating the safety and efficacy of Fabhalta, with key endpoints being the reduction of proteinuria and the decline in estimated glomerular filtration rate (eGFR) over a 24-month period.

In the interim analysis, which focused on proteinuria reduction, Fabhalta demonstrated a substantial 38% reduction in proteinuria compared to placebo.

 Particularly, the study included patients who were already on stable doses of renin-angiotensin system inhibitors, with or without sodium-glucose cotransporter-2.

 Fabhalta’s safety profile remained consistent with findings from previous studies, reinforcing its potential as a reliable treatment option.

Reflecting on this milestone, Victor Bultó, President of Novartis US, emphasized the significance of Fabhalta’s approval as a first-in-class medication for IgA nephropathy.

He described it as an important step in Novartis’s broader mission to transform care for rare renal diseases by providing new treatment options for patients in dire need.

 Bultó also reaffirmed the company’s dedication to partnering with the IgAN community as they continue advancing their extensive renal disease therapies portfolio.

Professor Dana Rizk, an Investigator and Member of the APPLAUSE-IgAN Steering Committee, highlighted the challenges posed by the heterogeneous and progressive nature of IgA nephropathy, which has historically made effective treatment difficult.

She noted that the growing body of clinical evidence highlighted the critical role of complement activation in IgAN. She expressed her satisfaction that this new targeted treatment option is now available to patients.

Adding to the conversation, Bonnie Schneider, Director and Co-Founder of the IgAN Foundation, remarked that the FDA’s approval of Fabhalta offers renewed hope for individuals living with IgA nephropathy.

She described the drug as a significant innovation in the treatment landscape, offering patients a new approach to managing this complex disease.

Schneider, who is also a parent of a son who has lived with IgAN for 20 years, shared her personal understanding of the fear and uncertainty that accompany an IgAN diagnosis and the profound impact it can have on patients and their families.

In addition to Fabhalta, Novartis is actively pursuing the development of two other IgAN therapies, atrasentan and zigakibart, which are currently in late-stage trials.

Atrasentan has already received FDA filing acceptance, while zigakibart is progressing through Phase III development, reaffirming Novartis’s commitment to addressing unmet needs in rare renal diseases.