Objective:

To report the FDA's traditional approval of iptacopan for slowing kidney function decline in adults with primary IgA nephropathy.

Approach:

• Approval Process: The approval was granted under priority review based on findings from the phase 3 APPLAUSE-IgAN trial.

Key Findings:

• Iptacopan (Fabhalta) previously received accelerated approval in August 2023 for reducing proteinuria.
• The annualized mean decline in estimated glomerular filtration rate was 3.0 mL/min/1.73 m² per year with iptacopan compared to 5.7 mL/min/1.73 m² per year with placebo.
• Reductions in proteinuria were observed as early as 2 weeks and sustained over the treatment period.

Interpretation:

The approval reinforces the importance of targeting underlying disease mechanisms, including complement activation, in treating IgAN.

Limitations:

• Iptacopan may increase the risk of serious infections caused by encapsulated bacteria.
• Available only through a Risk Evaluation and Mitigation Strategy program requiring appropriate vaccinations before treatment.

Conclusion:

The ability to significantly slow kidney function decline is a critical treatment goal.

Sources:

• Novartis Press Release