Novartis' Newly FDA-Approved Rare Blood Disorder Drug Aces Phase 3 Study In Ultra Rare Kidney Failure Disease

Novartis AG NVS announced topline results from the six-month, double-blind period of the Phase 3 APPEAR-C3G study of iptacopan for C3 glomerulopathy (C3G), a group of related conditions that cause the kidneys to malfunction.

Approximately 50% of C3G patients progress to kidney failure within ten years of diagnosis. Each year, approximately 1-2 people per million worldwide are newly diagnosed with C3G.

Last week, the FDA approved iptacopan, under the brand name Fabhalta, as the first oral monotherapy for a rare blood disorder, paroxysmal nocturnal hemoglobinuria.

The study met its primary endpoint, with iptacopan (200 mg twice daily) demonstrating superiority compared to placebo in providing clinically meaningful and statistically significant proteinuria (protein in urine) reduction on top of background therapy at six months. 

Also Read: Novartis Outlines Pure-Play Strategy Progress After Generics Business Spin-Off.

The safety profile of iptacopan was consistent with previously reported data.

Potential regulatory submissions are expected in 2024. The APPEAR-C3G study continues for a six-month, open-label period, in which all patients receive iptacopan, including those previously receiving a placebo. 

In addition, enrollment is ongoing in a separate cohort of adolescent patients with C3G.

Novartis also announced results from the extension period of the Phase 3 APPLY-PNH trial of oral monotherapy Fabhalta (iptacopan) in PNH patients who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy.

Continuous Fabhalta treatment (200 mg twice daily) for 48 weeks enabled sustained hemoglobin-level increases to near-normal (12 g/dL or more), blood transfusion avoidance, and reduced patient-reported fatigue in the majority of patients; comparable benefits emerged in those patients switching from anti-C5 therapy to Fabhalta in the extension.

Price Action: NVS shares are up 0.53% at $96.81 during the premarket session on the last check Monday.

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