FABHALTA

FABHALTA is an FDA-registered drug product whose active ingredient is IPTACOPAN. It is labelled by Novartis Pharmaceuticals Corporation taken by oral and marketed as a human prescription drug product. Identifiers on file: NDC 0078-1189, application NDA218276, label effective 20260716. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing IPTACOPAN are most often described in connection with: treatment adults, paroxysmal nocturnal, immunoglobulin nephropathy, complement glomerulopathy, fabhalta indicated, hemoglobinuria pnh, kidney function, igan risk. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

1 INDICATIONS AND USAGE FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) slowing kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. ( 1.2 ) 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 slow kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. 1.3 Complement 3 Glomerulopathy FABHALTA is indicated for the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria.

2 DOSAGE AND ADMINISTRATION 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. For patients switching from ravulizumab, initiate FABHALTA no later than 6 weeks after the last dose of ravulizumab. There is no available information regarding the timeframe for initiation of FABHALTA after other PNH therapies.

4 CONTRAINDICATIONS 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. Serious hypersensitivity to iptacopan or any of the excipients. ( 4 ) Initiation in patients with unresolved serious infection caused by encapsulated bacteria. ( 4 )

6 ADVERSE REACTIONS 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 abdominal pain and dizziness. ( 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. In APPLY-PNH, serious adverse reactions were reported in 2 (3%) patients with PNH receiving FABHALTA. Serious adverse reactions included pyelonephritis, urinary tract infection and COVID-19. In APPOINT-PNH, serious adverse reactions were reported in 2 (5%) patients with PNH receiving FABHALTA. Serious adverse reactions included COVID-19 and bacterial pneumonia. The most common adverse reactions (≥ 10%) with FABHALTA were headache, nasopharyngitis, diarrhea, abdominal pain, bacterial infection, viral infection, nausea, and rash. Table 1 describes the adverse reactions that occurred in > 5% of patients treated with FABHALTA in the APPLY-PNH or APPOINT-PNH studies. Table 1: Adverse Reactions Reported in > 5% of Patients Treated with FABHALTA in APPLY-PNH or APPOINT-PNH Studies (24-Week Treatment Period) a Includes similar terms. b Nasopharyngitis contains: rhinitis allergic, upper respiratory tract infection, pharyngitis, rhinitis. c Bacterial infection contains: pyelonephritis, urinary tract infection, bronchitis bacterial, bronchitis haemophilus, cholecystitis, folliculitis, cellulitis, arthritis bacterial, sepsis, klebsiella infection, staphylococcal infection, Pseudomonas infection, hordeolum, pneumonia bacterial. d Viral infection contains: COVID-19, herpes zoster, oral herpes, nasal herpes, influenza A virus test positive, influenza. e Lipid disorder contains: dyslipidemia, blood cholesterol increased, low density lipoprotein increased, hypercholesterolemia, blood triglycerides increased, hyperlipidemia. f Rash contains: dermatitis allergic, acne, erythema multiforme, rash maculo-papular, rash erythematous. Adverse reactions APPLY-PNH APPOINT-PNH FABHALTA (N = 62) n (%) Anti-C5 (Eculizumab or Ravulizumab) (N = 35) n (%) FABHALTA (N = 40) n (%) Headache a 12 (19) 1 (3) 11 (28) Nasopharyngitis b 10 (16) 6 (17) 6 (15) Diarrhea 9 (15) 2 (6) 3 (8) Abdominal pain a 9 (15) 1 (3) 3 (8) Bacterial infection c 7 (11) 4 (11) 2 (5) Nausea 6 (10) 1 (3) 2 (5) Viral infection d 6 (10) 11 (31) 7 (18) Arthralgia 5 (8) 1 (3) 0 Thrombocytopenia a 4 (6) 0 0 Dizziness 4 (6) 0 1 (3) Systemic hypertension a 4 (6) 0 0 Lipid disorder e 4 (6) 0 3 (8) Rash f 2 (3) 0 4 (10) Clinically relevant adverse reactions reported in less than or equal to 5% of patients includes urticaria in one patient (3%) in APPOINT-PNH. Description of Select Adverse Reactions (graded per NCI CTCAE Version 4.03 unless noted otherwise) Platelet Count Decreased Of the 37 FABHALTA-treated patients who had normal platelet counts at baseline in APPLY-PNH, 43% experienced any Grade thrombocytopenia during the randomized treatment period. Three FABHALTA-treated patients in APPLY-PNH experienced decreased platelets that worsened to Grade ≥ 3 from baseline (one patient with normal platelets that worsened to Grade 4, one patient with baseline Grade 1 that worsened to Grade 4, and one patient with baseline Grade 3 that worsened to Grade 4). Immunoglobulin A Nephropathy (IgAN) The safety of FABHALTA was evaluated in APPLAUSE-IgAN, a randomized placebo-controlled, double-blind clinical study in adults with IgAN (eGFR ≥ 20 mL/min/1.73 m 2 at baseline). The data below reflects FABHALTA exposure in 259 patients with IgAN (eGFR ≥ 20 mL/min/1.73 m 2 at baseline) with a median duration of 102 weeks (up to 107 weeks) in APPLAUSE-IgAN. Table 2 describes the adverse reactions that occurred in ≥ 3 % of patients treated with FABHALTA and were ≥ 2% higher in frequency than placebo. All of these adverse reactions were mild or moderate in severity. Table 2: Adverse Reactions Reported in ≥ 3% of Adult Patients with IgAN (eGFR ≥ 20 mL/min/1.73 m 2 ) Treated with FABHALTA and ≥ 2% Higher in Frequency Than Placebo in APPLAUSE-IgAN a Includes similar terms Adverse reaction FABHALTA (N = 259) n (%) Placebo (N = 258) n (%) Abdominal pain a 20 (8) 10 (4) Dizziness 13 (5) 6 (2) Nausea 11 (4) 2 (1) Serious infections caused by encapsulated bacteria reported in APPLAUSE-IgAN occurred in 3 patients (1.2%) on FABHALTA and none on placebo. Complement 3 Glomerulopathy (C3G) The safety of FABHALTA was evaluated in APPEAR-C3G, a randomized, placebo-controlled, double-blind trial in adult patients with native kidney C3G. No new adverse reactions were identified during the 6-month placebo-controlled period of APPEAR-C3G, in which 38 patients were treated with FABHALTA and 36 patients were treated with placebo. The most common adverse reactions that occurred in ≥ 10% of patients treated with FABHALTA and were ≥ 5% higher in frequency than placebo were nasopharyngitis (11% in FABHALTA, 3% placebo) and viral infections (29% in FABHALTA, 22% placebo), mainly respiratory infections. One patient (3%) on FABHALTA and none on placebo had a serious adverse reaction of pneumonia and bacteremia secondary to an encapsulated organism ( S. pneumoniae ).

5 FDA recall record(s) match this product name or manufacturer. A match means the firm or product name appears in the enforcement report; confirm lot numbers and dates against the official FDA notice before acting.

What is FABHALTA used for?
1 INDICATIONS AND USAGE FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). ( 1.1 ) slowing kidney function decline in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. ( 1.2 ) the treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria. ( 1.3 ) 1.1 …

Who makes FABHALTA?
Novartis Pharmaceuticals Corporation is the labeler of record for FABHALTA.

What form does FABHALTA come in?
administered by oral. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is FABHALTA prescription or over-the-counter?
The FDA label for this product is recorded as human prescription drug. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Has FABHALTA been recalled?
5 FDA recall record(s) match this product name or manufacturer. Review the recall and safety notices section on this page and confirm lot numbers against the official FDA enforcement report.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.