Iptacopan

Iptacopan binds 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, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization.

• 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.

• FABHALTA is contraindicated: in patients with serious hypersensitivity to iptacopan or any of the excipients. for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type b.contraindicated

200 mg orally twice daily with or without food. ( 2.2 ) 2.1 Recommended Vaccination and Prophylaxis for Encapsulated Bacterial Infections Vaccinate patients against encapsulated bacteria, including Streptococcus pneumoniae and Neisseria meningitidis (serogroups A, C, W, Y and B) , according to current ACIP recommendations at least 2 weeks prior to initiation of FABHALTA [see Warnings and Precautions (5.1)] . If urgent FABHALTA therapy is indicated in a patient who is not up to date with vaccines for Streptococcus pneumoniae and Neisseria meningitidis according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible [see Warnings and Precautions (5.1)] . Healthcare providers who prescribe FABHALTA must enroll in the FABHALTA REMS [see Warnings and Precautions (5.2)] . 2.2 Recommended Dosage The recommended dosage of FABHALTA is 200 mg orally twice daily without regard to food. Swallow capsules whole. Do not open, break, or chew capsules. If a dose or doses are missed, advise the patient to take one dose of FABHALTA as soon as possible (even if it is soon before the next scheduled dose) and then to resume the regular dosing schedule. 2.3 PNH Patients Switching From Anti-C5 (eculizumab, ravulizumab) to FABHALTA To reduce the potential risk of hemolysis with abrupt discontinuation of other PNH therapies: For patients switching from eculizumab, initiate FABHALTA no later than 1 week after the last dose of eculizumab.

Monitoring of PNH Manifestations After FABHALTA Discontinuation: Monitor for signs of hemolysis after discontinuation. ( 5.3 ) Hyperlipidemia: Monitor serum lipid parameters periodically during treatment and initiate cholesterol-lowering medication, if indicated. ( 5.4 ) 5.1 Serious Infections Caused by Encapsulated Bacteria FABHALTA, a complement inhibitor, increases a patient’s susceptibility to serious, life-threatening, or fatal infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis (caused by any serogroup, including non-groupable strains), and Haemophilus influenzae type b. Life-threatening and fatal infections with encapsulated bacteria have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of FABHALTA treatment is contraindicated in patients with unresolved serious infections caused by encapsulated bacteria. Complete or update vaccination against encapsulated bacteria at least 2 weeks prior to administration of the first dose of FABHALTA, according to the current ACIP recommendations for patients receiving a complement inhibitor. Revaccinate patients in accordance with ACIP recommendations considering the duration of therapy with FABHALTA. Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the administration schedule in the vaccine prescribing information.

The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling: Serious Infections Caused by Encapsulated Bacteria [see Warnings and Precautions (5.1)] . Hyperlipidemia [see Warnings and Precautions (5.4)] . Most common adverse reactions in adults with PNH (incidence ≥ 10%) were headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, viral infection, nausea and rash. ( 6.1 ) Most common adverse reactions in adults with IgAN (incidence ≥ 5%) were upper respiratory tract infection, lipid disorder, and abdominal pain. ( 6.1 ) Most common adverse reactions in adults with C3G (incidence ≥ 10%) were nasopharyngitis and viral infections. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Novartis Pharmaceuticals Corporation at 1-888-669-6682 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Paroxysmal Nocturnal Hemoglobinuria (PNH) The data described below reflects the exposure in adults with PNH who received FABHALTA (n = 62) or anti-C5 treatment (US-approved and non-US-approved eculizumab product or US-approved and non-US-approved ravulizumab product, n = 35) in APPLY-PNH [NCT04558918] and adults who received FABHALTA (n = 40) in APPOINT-PNH [NCT04820530] at the recommended dosing regimen for 24 weeks.

Severe hepatic impairment: Use not recommended. ( 8.7 ) 8.1 Pregnancy Risk Summary Available data from clinical trials with FABHALTA use in pregnant women are insufficient to identify a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. There are risks to the mother and fetus associated with untreated PNH, IgAN, or C3G in pregnancy (see Clinical Considerations) . The use of FABHALTA in pregnant women or women planning to become pregnant may be considered following an assessment of the risks and benefits. In animal reproduction studies, oral administration of iptacopan to pregnant rats and rabbits during organogenesis at exposures 4- to 6-times the human exposure (based on AUC) at the maximum recommended human dose (MRHD) of 200 mg twice daily did not induce embryo or fetal toxicity (see Data) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. All pregnancies have a background risk of major birth defects, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriages in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

Absorption Following oral administration, iptacopan reached peak plasma concentrations approximately 2 hours post dose. At the recommended dosing regimen of 200 mg twice daily, steady state is achieved in approximately 5 days with minor accumulation (1.4-fold).

• Dec 5, 2023NDANDA218276Novartis

1. 1Headache8212%
2. 2Fatigue568.4%
3. 3Nausea436.5%
4. 4Pyrexia436.5%
5. 5Diarrhoea406.0%
6. 6Haemoglobin Decreased406.0%
7. 7Product Dose Omission Issue355.3%
8. 8Drug Ineffective324.8%
9. 9Breakthrough Haemolysis314.7%
10. 10Rash304.5%
11. 11Off Label Use294.4%
12. 12Dyspnoea263.9%
13. 13Platelet Count Decreased263.9%
14. 14Vomiting253.8%
15. 15Asthenia243.6%

The 10 most recently updated of 38 ClinicalTrials.gov registrations naming Iptacopan as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
  Recruiting · Phase 3 · Interventional · 65 enrolled · Novartis Pharmaceuticals
  NCT05795140updated 2026-06-04
• Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN
  Recruiting · Phase 3 · Interventional · 31 enrolled · Novartis Pharmaceuticals
  NCT06994845updated 2026-06-01
• A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy
  Active not recruiting · Phase 3 · Interventional · 448 enrolled · Novartis Pharmaceuticals
  NCT04557462updated 2026-06-01
• A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
  Recruiting · Observational · 80 enrolled · Novartis Pharmaceuticals
  NCT06931691updated 2026-06-01
• Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN
  Recruiting · Phase 3 · Interventional · 106 enrolled · Novartis Pharmaceuticals
  NCT05755386updated 2026-06-01
• Fabhalta Capsules Specified Drug-use Survey
  Recruiting · Observational · 50 enrolled · Novartis Pharmaceuticals
  NCT07156149updated 2026-06-01
• Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
  Completed · Phase 3 · Interventional · 34 enrolled · Novartis Pharmaceuticals
  NCT04889430updated 2026-06-01
• Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V
  Recruiting · Phase 2 · Interventional · 240 enrolled · Novartis Pharmaceuticals
  NCT05268289updated 2026-06-01
• Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
  Recruiting · Phase 3 · Interventional · 98 enrolled · Novartis Pharmaceuticals
  NCT04817618updated 2026-06-01
• A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
  Recruiting · Phase 3 · Interventional · 146 enrolled · Novartis Pharmaceuticals
  NCT06517758updated 2026-05-27

Frequently asked questions

How does Iptacopan work?

Iptacopan binds 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, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization.

What is Iptacopan used for?

According to FDA labeling, Iptacopan carries indications including: 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.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Iptacopan?

Iptacopan is classified as Complement inhibitors, Complement Factor B Inhibitor, Complement Factor B Inhibitors, Complement Inhibitors, Decreased Complement Production.

What are the brand names for Iptacopan?

Iptacopan is marketed under brand names including Fabhalta.

What are the contraindications for Iptacopan?

Iptacopan labeling lists contraindications including: FABHALTA is contraindicated: in patients with serious hypersensitivity to iptacopan or any of the excipients. for initiation in patients with unresolved serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type b.. Always consult the full prescribing information and a clinician.