Switzerland-based multinational pharmaceutical corporation Novartis, announced on Thursday that it has received approval from the US Food and Drug Administration for oral Fabhalta (iptacopan), the first and only treatment approved in C3 glomerulopathy (C3G) to reduce proteinuria, a progressive and ultra-rare kidney disease that, until now, has had no approved treatments.

Fabhalta is the only oral inhibitor of the alternative complement pathway to selectively target what is thought to be the underlying cause of the disease. Before the approval of Fabhalta, patients had to rely on supportive care, broad immunosuppression, and symptom management.

The pivotal Phase III APPEAR-C3G study evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G1,10. The study was comprised of a 6-month randomised, double-blind treatment period with Fabhalta compared to placebo in addition to supportive care, followed by an additional 6-month open-label treatment period where all participants received Fabhalta.

Victor Bulto, president US, Novartis, said, 'We extend our deepest gratitude to the patients and investigators who participated in our clinical trials, without whom this first FDA approval in C3G wouldn't have been possible. With this additional approval for Fabhalta – the second in kidney disease – we will leverage our established capabilities and expertise to bring this innovative treatment to patients in need as we work to help transform care for people living with kidney diseases.'