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Novartis NVS announced positive top-line results from a late-stage study on Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway.
APPULSE-PNH is a phase IIIB multicenter, single-arm, open-label study to evaluate the efficacy and safety of twice-daily oral Fabhalta monotherapy (200mg) in adults with PNH who were switched from anti-C5 therapies (eculizumab or ravulizumab).
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More on NVS’ Study of Fabhalta
The APPULSE-PNH study enrolled 52 participants who received Fabhalta for 24 weeks.
In the Phase IIIB APPULSE-PNH study, oral Fabhalta improved the average hemoglobin (Hb) level versus baseline in adult patients with PNH who were switched from anti-C5 therapies.
The positive results reinforce Fabhalta's ability to benefit both patients previously treated with anti-C5 therapies studied in the APPULSE-PNH and APPLY-PNH trials and complement-inhibitor naïve patients studied in the APPOINT-PNH trial.
The safety profile of Fabhalta monotherapy was consistent with previously reported data.
Fabhalta obtained FDA approval in December 2023 for the treatment of adults with PNH. It also received the European Medicines Agency’s approval in May 2024 for the treatment of adults with PNH with hemolytic anemia.
We remind investors that the FDA recently granted Fabhalta accelerated approval for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression.
Fabhalta is being evaluated in a broad range of rare kidney diseases, including C3 glomerulopathy, atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis and lupus nephritis.
NVS Presents Long-Term Data on Scemblix
Novartis also announced positive, longer-term results from the phase III ASC4FIRST study on leukemia drug Scemblix (asciminib).
Scemblix demonstrated sustained superior major molecular response (MMR) versus all investigator-selected tyrosine kinase inhibitors (TKIs) and versus imatinib alone. Scemblix met both key secondary endpoints at the end of 96 weeks.
Scemblix showed a clinically relevant 15.1% higher MMR rate versus second-generation TKIs (72.0% versus 56.9%).
NVS Looks to Strengthen Pipeline
NVS is a pure-play innovative medicine company with a focus on core therapeutic areas — cardiovascular, renal and metabolic, immunology, neuroscience and oncology.