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🏅 FDA Orphan Designation

FABHALTA

IPTACOPAN

Manufacturer: Novartis Pharmaceuticals Corporation

Indicated for:
C3 glomerulopathyOrphanParoxysmal nocturnal hemoglobinuriaOrphanImmunoglobulin A nephropathyAccel.IgA NephropathyAccel.
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Black Box Warning (Boxed Warning)

A Boxed Warning is the FDA's strongest safety warning — it indicates serious or life-threatening risks identified during clinical trials or post-market surveillance. It does not mean the medication is unsafe for all patients; your prescriber weighs these risks against the benefits for your specific situation. Source: FDA-approved prescribing information.

View full prescribing information on DailyMed (NLM) ↗

FDA-Approved Indications (4)

C3 glomerulopathyOrphan Designation

treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria

1 INDICATIONS AND USAGE FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) the reduction of proteinuria in adul

Immunoglobulin A nephropathyAccelerated Approval

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

Population: adults

IgA NephropathyAccelerated Approval

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

Indications & Usage

1 INDICATIONS AND USAGE FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) 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. ( 1.2 ) 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. the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria. ( 1.3 ) 1.1 Paroxysmal Nocturnal Hemoglobinuria FABHALTA is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). 1.2 Immunoglobulin A Nephropathy FABHALTA is indicated to reduce 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. 1.3 Complement 3 Glomerulopathy FABHALTA is indicated for the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria.

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Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.