Fabhalta FDA Approval History

Last updated by Judith Stewart, BPharm on Aug 15, 2024.

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, Primary Immunoglobulin A Nephropathy

Fabhalta (iptacopan) is a complement factor B inhibitor used for the treatment of paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.

• Fabhalta is indicated for:
  - the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
  - the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g.
  This indication is approved under accelerated approval based on reduction of proteinuria. It has not been established whether Fabhalta slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. 
• 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.
  IgAN is a progressive, rare disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria.
• 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 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.
• Fabhalta works by binding to factor B of the alternative complement pathway and regulates the cleavage of C3, generation of downstream effectors, and the amplification of the terminal pathway.
  - in PNH, Fabhalta acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis.
  - in IgAN, Fabhalta binds to factor B and inhibits the effect of the alternative pathway which is thought to contribute to the pathogenesis of the condition.
• 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:
  - in patients with PNH include headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, viral infection, nausea and rash.
  - in patients with IgAN include upper respiratory tract infection, lipid disorder, and abdominal pain.
• Iptacopan is also being investigated for the treatment of glomerulopathy, atypical hemolytic uremic syndrome, and immune complex membranoproliferative glomerulonephritis.

Development timeline for Fabhalta

Further information

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