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Fabhalta (IPTACOPAN)
Iptacopan binds to Factor B, regulating C3 cleavage and downstream effects in the alternative complement pathway, controlling IVH and EVH in PNH and inhibiting local activation in IgAN and C3G.
Fabhalta (iptacopan) is a small molecule drug developed by Novartis, currently owned by the same company. It was FDA-approved in 2023 for the treatment of Paroxysmal Nocturnal Hemoglobinuria, Primary Immunoglobulin A Nephropathy, and Complement 3 Glomerulopathy. The commercial status of Fabhalta is patented, with no generic manufacturers available. Key safety considerations are not specified. As a small molecule, Fabhalta works by modulating the complement system, which plays a crucial role in the body's immune response.
At a glance
| Generic name | IPTACOPAN |
|---|---|
| Sponsor | Novartis |
| Target | Factor B |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | FDA-approved |
| First approval | 2023 |
Mechanism of action
Iptacopan works by binding to Factor B in the alternative complement pathway. This binding regulates the cleavage of C3 and the generation of downstream effectors, which helps control both intravascular and extravascular hemolysis in PNH, and inhibits the local activation of the alternative pathway in conditions like IgAN and C3G.
Approved indications
- Paroxysmal Nocturnal Hemoglobinuria
- Primary Immunoglobulin A Nephropathy
- Complement 3 Glomerulopathy
Boxed warnings
- 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 least 2 weeks prior to the first dose of FABHALTA, unless the risks of delaying 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. ( 5.1 ) 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. ( 5.1 ) FABHALTA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called FABHALTA REMS. ( 5.2 )
Common side effects
- headache
- nasopharyngitis
- diarrhea
- abdominal pain
- bacterial infection
- nausea
- viral infection
- arthralgia
- thrombocytopenia
- dizziness
- systemic hypertension
- lipid disorder
Drug interactions
- rifampin
- gemfibrozil
Key clinical trials
- Iptacopan in Patients With ANCA Associated Vasculitis (PHASE2)
- A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG. (PHASE3)
- Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN (PHASE2)
- Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V (PHASE2)
- IgA Nephropathy Insights From Treatment Experience Among Patients Receiving Iptacopan and/or Atrasentan Using Primary Data Collection
- Fabhalta Capsules Specified Drug-use Survey
- Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (PHASE3)
- Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN (PHASE3)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Fabhalta CI brief — competitive landscape report
- Fabhalta updates RSS · CI watch RSS
- Novartis portfolio CI