Novartis claims Fabhalta transforms lives of PNH patients with improved hemoglobin

In a significant development for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), Novartis AG announced the topline results from its APPULSE-PNH Phase 3B study of Fabhalta (iptacopan). This study focused on adult patients who were switched from anti-C5 therapies, specifically those whose hemoglobin levels remained above 10 g/dL following treatment with eculizumab or ravulizumab. The results indicated an improvement in average hemoglobin levels after 24 weeks of treatment with Fabhalta, suggesting its efficacy in managing this rare blood disorder. This news, reported on a Friday in early December 2024, marks a notable step in Novartis’s ongoing efforts to address complement-mediated diseases. The safety profile of Fabhalta monotherapy was consistent with previously reported data, providing reassurance regarding its overall tolerability for patients. Furthermore, this Phase 3B study's findings will be shared at a prominent medical meeting scheduled for 2025, highlighting the importance of these results in the broader clinical context. The Company recently received accelerated approval from the FDA for Fabhalta, specifically to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of rapid disease progression. This sets a promising precedent for Novartis’s continued investment in complement-mediated diseases and diversifies their treatment options in an evolving therapeutic landscape. In addition to these advancements, Novartis has raised its mid-term sales guidance. Their compound annual growth rate (CAGR) target for 2023-2028 has been increased to 6%, reflecting confidence in the current growth drivers and anticipated launches. This revision is largely based on favorable performance metrics, which should maintain U.S. exclusivity well into the 2030s. The overall sentiment among analysts remains neutral, with expectations of a slight decrease in stock value noted in recent evaluations. Novartis’s stock has experienced fluctuations recently, having fallen by 12.4% over the past three months, which is indicative of changing market perceptions and confidence in its business model. The advancement and positive findings regarding Fabhalta could influence future performance as the company navigates potential market growth and challenges ahead.