FABHALTA (iptacopan hydrochloride)
FABHALTA is indicated for the treatment of Paroxysmal Nocturnal Hemoglobinuria; Immunoglobulin A Nephropathy; Complement 3 Glomerulopathy.
How FABHALTA Works
Iptacopan binds to Factor B of the alternative complement pathway, proximally regulating the cleavage of C3 and the amplification of the terminal pathway. In PNH, this proximal inhibition allows for the control of both C3b-mediated extravascular hemolysis (EVH) and terminal complement-mediated intravascular hemolysis (IVH). In IgAN and C3G, Fabhalta inhibits the overactivation of the alternative pathway, reducing C3 deposition and the subsequent inflammatory processes that lead to glomerular damage and proteinuria.
Details
- Status
- Prescription
- First Approved
- 2023-12-05
- Patent Cliff
- 2041
- Revenue
- $181M (Q4-2025)
- Routes
- ORAL
- Dosage Forms
- CAPSULE
FABHALTA Approval History
What FABHALTA Treats
3 indicationsFABHALTA is approved for 3 conditions since its original approval in 2023. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Paroxysmal Nocturnal Hemoglobinuria
- Immunoglobulin A Nephropathy
- Complement 3 Glomerulopathy
FABHALTA Boxed Warning
SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA FABHALTA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b [see Warnings and Precautions (5.1)] . Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if ...
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA FABHALTA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b [see Warnings and Precautions (5.1)] . Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of FABHALTA, unless the risks of delaying therapy with FABHALTA outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by encapsulated bacteria. Patients receiving FABHALTA are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, FABHALTA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the FABHALTA REMS [see Warnings and Precautions (5.2)] . WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA See full prescribing information for complete boxed warning. FABHALTA increases the risk of serious and life-threatening infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b. Complete or update vaccination for encapsulated bacteria at
FABHALTA Competitive Set
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Indication competitors
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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
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Active Pipeline
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Key Completed Trials
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Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
FABHALTA FDA Label Details
Indications & Usage
FDA Label (PDF)FABHALTA is indicated for the treatment of Paroxysmal Nocturnal Hemoglobinuria; Immunoglobulin A Nephropathy; Complement 3 Glomerulopathy.
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA FABHALTA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type b [see Warnings and Prec...
FABHALTA Patents & Exclusivity
Patents (8 active)
Exclusivity
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Revenue Insights
- • Q4-2025: $181M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2041
- • 10 active patents
Trial Analysis
- • Clinical trial tracking
- • Development stage analysis
Competitive Landscape
- • 9 similar drugs
- • Same target/indication analysis
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.