Fabhalta FDA Approval History
Last updated by Judith Stewart, BPharm on Dec 7, 2023.
FDA Approved: Yes (First approved December 5, 2023)
Brand name: Fabhalta
Generic name: iptacopan
Dosage form: Capsules
Company: Novartis Pharmaceuticals Corporation
Treatment for: Paroxysmal Nocturnal Hemoglobinuria
Fabhalta (iptacopan) is a first-in-class, complement factor B inhibitor indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria.
- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function, resulting in anemia, fatigue, and other debilitating symptoms.
- Current treatments for patients with PNH include the complement C5 inhibitors eculizumab and ravulizumab which prevent intravascular hemolysis, and the complement C3 inhibitor pegcetacoplan which prevents both intravascular and extravascular hemolysis.
- Fabhalta contains iptacopan which is a targeted factor B inhibitor of the alternative complement pathway that acts upstream of the C5 terminal pathway to prevent both intravascular and extravascular hemolysis in PNH.
- FDA approval of Fabhalta was based on the results of the APPLY-PNH trial in adults with PNH and anemia despite prior anti-C5 treatment, and supported by the APPOINT-PNH study in complement inhibitor-naïve patients.
- In the APPLY-PNH trial, patients who switched to Fabhalta experienced superior increases of hemoglobin levels ≥ 2 g/dL (82.3% vs. 0%) and hemoglobin level ≥ 12 g/dL (67.7% vs. 0%), both in the absence of red blood cell transfusions, vs. patients who continued on anti-C5 treatment. - Fabhalta capsules are taken twice daily with or without food.
- The Fabhalta product label carries a boxed warning for an increased risk of serious and life-threatening infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B.
- Warnings and precautions associated with Fabhalta include hemolysis after discontinuation and hyperlipidemia.
- Common adverse reactions include headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, viral infection, nausea and rash.
- Iptacopan is also being investigated in Phase III studies for IgA nephropathy (APPLAUSE-IgAN [NCT04578834], C3 glomerulopathy (APPEAR-C3G [NCT04817618]), atypical hemolytic uremic syndrome (APPELHUS [NCT04889430]) and immune complex membranoproliferative glomerulonephritis (APPARENT [NCT05755386]).
Development timeline for Fabhalta
Further information
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