FDA Grants Landmark Approval for C3G Treatment

On March 20, Novartis announced that its oral therapy Fabhalta (iptacopan) has been approved by the U.S. Food and Drug Administration (FDA) as the first and only treatment for C3 glomerulopathy (C3G), a rare and progressive kidney disease. This innovative complement inhibitor works by specifically targeting the root mechanism of C3G—overactivation of the alternative complement pathway.

Clinical Evidence Supports Disease Modification

Approval was based on pivotal clinical trials that showed Fabhalta significantly reduced proteinuria and stabilized renal function over 12 months in adult C3G patients, compared to placebo. The once-daily oral regimen was well-tolerated, with adverse events consistent with the known safety profile of complement inhibitors.

Expert and Patient Community Reactions

Nephrologists and patient advocacy groups welcomed the decision, highlighting that Fabhalta addresses a long-standing unmet need in this population. Previous treatments for C3G offered only supportive management, whereas Fabhalta demonstrates true disease-modifying potential.

Outlook: Transforming Rare Kidney Disease Care

Novartis plans a rapid launch and expanded access program alongside further research into Fabhalta’s benefits in related complement-driven disorders. Analysts indicate this approval positions Novartis as a leader in renal rare disease innovation and sets a precedent for oral complement therapies in nephrology.