FDA

Fabhalta approved for C3 glomerulopathy

March 25, 2025

FDA approved Fabhalta (iptocopan) for the treatment of adults with C3 glomerulopathy (C3G), to reduce proteinuria.

Fabhalta is also indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.

Efficacy

Approval of the new indication was based on data from the phase 3 APPEAR-C3G study (NCT04817618) that evaluated the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G. The study was comprised of a 6-month randomized, double-blind treatment period with Fabhalta compared with placebo in addition to supportive care, followed by a 6-month open-label treatment period during which all participants received Fabhalta. Patients were required to be on a maximally tolerated renin-angiotensin system inhibitor and could be on a corticosteroid and/or mycophenolate mofetil/sodium at baseline.

Treatment with Fabhalta resulted in clinically meaningful proteinuria reduction, which was seen as early as 14 days and sustained at 12 months. In the open-label period, proteinuria reduction was also seen in participants who switched to Fabhalta.

Safety

Fabhalta demonstrated a favorable safety profile, with no new safety signals. In patients with C3G, the most common adverse reactions (≥10%) with Fabhalta were nasopharyngitis and viral infections. Fabhalta may cause serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b. The drug is available only through a Risk Evaluation and Mitigation Strategy (REMS) that requires specific vaccinations.

Sources:

Novartis. Fabhalta (iptacopan) [package insert]. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/218276s004lbl.pdf Revised March 2025. Accessed March 24, 2025.

Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G). [News release]. Novartis. 2025. https://www.novartis.com/news/media-releases/novartis-receives-third-fda-approval-oral-fabhalta-iptacopan-first-and-only-treatment-approved-c3-glomerulopathy-c3g