Fabhalta
iptacopan · Capsule · Oral
Uses
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.
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.
Dosage forms and strengths
Capsules: 200 mg of iptacopan in pale yellow, opaque, hard gelatin capsules imprinted with “LNP200” on the body and “NVR” on the cap. Capsules: 200 mg ( 3 )
Contraindications
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 )
Warnings and precautions
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. If urgent FABHALTA therapy is indicated in a patient who is not up to date with vaccines against encapsulated bacteria according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer these vaccines as soon as possible. Various durations and regimens of antibacterial drug prophylaxis have been considered, but the optimal durations and drug regimens for prophylaxis and their efficacy have not been studied in unvaccinated or vaccinated patients receiving complement inhibitors, including FABHALTA. The benefits and risks of treatment with FABHALTA, as well as the benefits and risks of antibacterial drug prophylaxis in unvaccinated or vaccinated patients, must be considered against the known risks for serious infections caused by encapsulated bacteria. Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite development of antibodies following vaccination. Closely monitor patients for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected. Inform patients of these signs and symptoms and instruct patients to seek immediate medical care if these signs and symptoms occur. Promptly treat known infections. Serious infection may become rapidly life-threatening or fatal if not recognized and treated early. Consider interruption of FABHALTA in patients who are undergoing treatment for serious infections, depending on the risks of interrupting treatment in the disease being treated. FABHALTA is available only through a restricted program under a REMS [see Warnings and Precautions (5.2)] . 5.2 FABHALTA REMS FABHALTA is available only through a restricted program under a REMS called FABHALTA REMS, because of the risk of serious infections caused by encapsulated bacteria [see Warnings and Precautions (5.1)] . Notable requirements of the FABHALTA REMS include the following: Prescribers must enroll in the REMS. Prescribers must counsel patients about the risk of serious infections caused by encapsulated bacteria. Prescribers must provide patients with the REMS educational materials. Prescribers must assess patient vaccination status for vaccines against encapsulated bacteria and vaccinate if needed according to current ACIP recommendations two weeks prior to the first dose of FABHALTA. Prescribers must provide a prescription for antibacterial drug prophylaxis if treatment must be started urgently, and the patient is not up to date with vaccines against encapsulated bacteria according to current ACIP recommendations at least two weeks prior to the first dose of FABHALTA. Pharmacies that dispense FABHALTA must be certified in the FABHALTA REMS and must verify prescribers are certified. Patients must receive counseling from the prescriber about the need to receive vaccinations against encapsulated bacteria per ACIP recommendations, the need to take antibiotics as directed by the prescriber, and the early signs and symptoms of serious infections. Patients must be instructed to carry the Patient Safety Card with them at all times during treatment and for 2 weeks following the last dose of FABHALTA. Further information is available by telephone: 1-833-99FABHA (1-833-993-2242) or online at www.FABHALTA-REMS.com . 5.3 Monitoring of PNH Manifestations After FABHALTA Discontinuation In PNH patients, after discontinuing treatment with FABHALTA, closely monitor patients for at least 2 weeks after the last dose for signs and symptoms of hemolysis. These signs include elevated lactate dehydrogenase (LDH) levels along with a sudden decrease in hemoglobin or PNH clone size, fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (such as thrombosis, stroke and myocardial infarction), dysphagia, or erectile dysfunction. If discontinuation of FABHALTA is necessary, consider alternative therapy. If hemolysis occurs after discontinuation of FABHALTA, consider restarting treatment with FABHALTA, if appropriate, or initiating another treatment for PNH. 5.4 Hyperlipidemia FABHALTA may increase total cholesterol, LDL-cholesterol, and serum triglycerides [see Adverse Reactions (6.1)] . Of the 54 FABHALTA-treated patients who had a normal total cholesterol level at baseline in APPLY-PNH, 43% developed Grade 1 hypercholesterolemia during the randomized treatment period. One FABHALTA-treated patient in APPLY-PNH experienced increased total cholesterol that worsened to Grade 2 from Grade 1 at baseline.
Side effects
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.
Drug interactions
7 DRUG INTERACTIONS CYP2C8 inducers (e.g., rifampin): May decrease iptacopan exposure. Monitor for loss of efficacy. ( 7.1 ) Strong CYP2C8 inhibitors (e.g., gemfibrozil): May increase iptacopan exposure. Coadministration not recommended. ( 7.2 ) 7.1 CYP2C8 Inducers Concomitant use of CYP2C8 inducers (e.g., rifampin) may decrease iptacopan exposure, which may result in loss of or reduced efficacy of FABHALTA. Monitor the clinical response and discontinue use of the CYP2C8 inducer if loss of efficacy of FABHALTA is evident. 7.2 Strong CYP2C8 Inhibitors Concomitant use of strong CYP2C8 inhibitors (e.g., gemfibrozil) may increase iptacopan exposure, which may result in an increased risk for adverse reactions with FABHALTA. Coadministration with a strong CYP2C8 inhibitor is not recommended.
Use in specific populations
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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombosis, infections, bleeding, miscarriages, increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. IgAN in pregnancy is associated with adverse maternal outcomes, including increased rates of cesarean section, pregnancy-induced hypertension, pre-eclampsia and preterm delivery, and adverse fetal/neonatal outcomes, including stillbirth and low birth weight. C3G in pregnancy may be associated with adverse maternal outcomes, in particular preeclampsia and miscarriage, as well as adverse fetal outcomes including prematurity and low birth weight. Data Animal Data In an embryo-fetal development study in rats, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the MRHD based on AUC. In an embryo-fetal development study in rabbits, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 450 mg/kg/day, which corresponds to 6-times the MRHD based on AUC. In a pre- and postnatal development study in rats, oral administration of iptacopan during gestation, parturition, and lactation did not cause adverse effects in offspring when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the MRHD based on AUC. 8.2 Lactation Risk Summary There are no data on the presence of iptacopan or its metabolites in either human or animal milk, the effects on the breastfed child or on milk production. Since many medicinal products are secreted into human milk, and because of the potential for serious adverse reactions in a breastfed child, breastfeeding should be discontinued during treatment and for 5 days after the final dose. 8.4 Pediatric Use Safety and effectiveness in pediatric patients with PNH, IgAN, or C3G have not been established. 8.5 Geriatric Use There were 29 PNH patients 65 years of age and older in APPLY-PNH and APPOINT-PNH [see Clinical Studies (14)] . Of the total number of FABHALTA-treated patients during the 24-week treatment period in these studies, 21 (20.6%) were 65 years of age and older, while 7 (6.9%) were 75 years of age and older. There were 14 IgAN patients 65 years of age and older in APPLAUSE-IgAN [see Clinical Studies (14)] . Of the total number of FABHALTA-treated patients, 5 (2.1%) were 65 years of age and older. Clinical studies of FABHALTA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger patients. 8.7 Hepatic Impairment The use of FABHALTA is not recommended in patients with severe hepatic impairment (Child-Pugh class C). No dose adjustment is required for patients with mild (Child-Pugh class A) or moderate (Child-Pugh class B) hepatic impairment [see Clinical Pharmacology (12.3)] .
Pregnancy
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. Clinical Considerations Disease-Associated Maternal and/or Embryo/Fetal Risk PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombosis, infections, bleeding, miscarriages, increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery. IgAN in pregnancy is associated with adverse maternal outcomes, including increased rates of cesarean section, pregnancy-induced hypertension, pre-eclampsia and preterm delivery, and adverse fetal/neonatal outcomes, including stillbirth and low birth weight. C3G in pregnancy may be associated with adverse maternal outcomes, in particular preeclampsia and miscarriage, as well as adverse fetal outcomes including prematurity and low birth weight. Data Animal Data In an embryo-fetal development study in rats, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the MRHD based on AUC. In an embryo-fetal development study in rabbits, oral administration of iptacopan during organogenesis did not cause embryo-fetal toxicity when given up to the highest dose of 450 mg/kg/day, which corresponds to 6-times the MRHD based on AUC. In a pre- and postnatal development study in rats, oral administration of iptacopan during gestation, parturition, and lactation did not cause adverse effects in offspring when given up to the highest dose of 1,000 mg/kg/day, which corresponds to 4-times the MRHD based on AUC.
Pediatric use
8.4 Pediatric Use Safety and effectiveness in pediatric patients with PNH, IgAN, or C3G have not been established.
Geriatric use
8.5 Geriatric Use There were 29 PNH patients 65 years of age and older in APPLY-PNH and APPOINT-PNH [see Clinical Studies (14)] . Of the total number of FABHALTA-treated patients during the 24-week treatment period in these studies, 21 (20.6%) were 65 years of age and older, while 7 (6.9%) were 75 years of age and older. There were 14 IgAN patients 65 years of age and older in APPLAUSE-IgAN [see Clinical Studies (14)] . Of the total number of FABHALTA-treated patients, 5 (2.1%) were 65 years of age and older. Clinical studies of FABHALTA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger patients.
Description
11 DESCRIPTION FABHALTA contains iptacopan, a complement Factor B inhibitor. The molecular weight of iptacopan hydrochloride monohydrate is approximately 477 g/mol. The chemical name is (2 S ,4 S )-2-(4-Carboxyphenyl)-4-ethoxy-1-[(5-methoxy-7-methyl-1 H -indol-4-yl)methyl]piperidin-1-ium chloride―water (1/1). The molecular formula is C 25 H 30 N 2 O 4 ·HCl H 2 O. The structure is shown below. Iptacopan hydrochloride monohydrate is a white or almost white to pale purplish-pink powder. FABHALTA is supplied as hard gelatin capsules for oral administration. The capsules are packaged in high-density polyethylene (HDPE) bottles with induction seals and child resistant caps. Each FABHALTA capsule contains 200 mg iptacopan (provided as 225.8 mg iptacopan hydrochloride monohydrate) and the capsule shell contains the following inactive ingredients: gelatin, red ferric oxide, titanium dioxide, yellow ferric oxide. The black printing ink contains ferrosoferric oxide, potassium hydroxide, propylene glycol, shellac, and strong ammonia solution. chemical structure of iptacopan hydrochloride monohydrate
Mechanism of action
12.1 Mechanism of Action 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. Iptacopan acts proximally in the alternative pathway of the complement cascade to control both C3b-mediated EVH and terminal complement-mediated IVH. In IgAN, the deposition of galactose deficient IgA1 (Gd-IgA1) containing immune complexes in the kidney locally activates the alternative complement pathway which is thought to contribute to the pathogenesis of IgAN. By binding to Factor B, iptacopan inhibits the alternative pathway. In C3G, overactivation of the alternative complement pathway leads to C3 cleavage within the glomeruli resulting in C3 deposition and inflammation, which are thought to contribute to the pathogenesis of C3G. By binding to Factor B, iptacopan inhibits the alternative pathway.
How supplied
200 mg capsules: pale yellow opaque hard capsules, imprinted with “LNP200” on one half and “NVR” on the other half, packaged in a high-density polyethylene (HDPE) bottle with induction seal and child-resistant cap. Each bottle contains 60 capsules (NDC 0078-1189-20). Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (59°F and 86°F) [see USP Controlled Room Temperature].
Patient information
Advise the patient to read the FDA-approved patient labeling (Medication Guide). Serious Infections Caused by Encapsulated Bacteria Advise patients of the risk of serious infection. Inform patients of the need to complete or update their vaccinations against encapsulated bacteria at least 2 weeks prior to receiving the first dose of FABHALTA or to receive antibacterial drug prophylaxis if FABHALTA treatment must be initiated immediately and they have not been previously vaccinated. Inform patients of the requirement to be revaccinated according to current ACIP recommendations for encapsulated bacteria while on FABHALTA therapy [see Warnings and Precautions (5.1)] . Inform patients that vaccination may not prevent serious infection and to seek immediate medical attention if the following signs or symptoms occur [see Warnings and Precautions (5.1)] : fever with or without shivers or chills fever and a rash fever with chest pain and cough fever with breathlessness/fast breathing fever with high heart rate headache with nausea or vomiting headache and a fever headache with a stiff neck or stiff back confusion body aches with flu-like symptoms clammy skin eyes sensitive to light Inform patients that they will be given a Patient Safety Card for FABHALTA that they should carry with them at all times during and for 2 weeks following treatment with FABHALTA. This card describes symptoms which, if experienced, should prompt the patient to seek immediate medical evaluation. FABHALTA REMS FABHALTA is available only through a restricted program called FABHALTA REMS [see Warnings and Precautions (5.2)] . Inform the patient of the following notable requirements: Patients must receive counseling about the risk of serious infections caused by encapsulated bacteria. Patients must receive written educational materials about this risk. Patients must be instructed to carry the Patient Safety Card with them at all times during and for 2 weeks following treatment with FABHALTA. Patients must be instructed to complete or update vaccines against encapsulated bacteria per ACIP recommendations as directed by the prescriber prior to treatment with FABHALTA. Patients must receive antibiotics as directed by the prescriber if they are not up to date on vaccinations against encapsulated bacteria and have to start FABHALTA right away. Importance of Adherence to Dosing Schedule Inform patients with PNH of the importance of taking FABHALTA as prescribed in order to minimize the risk of hemolysis. Discontinuation Inform patients with PNH that they may develop serious hemolysis due to PNH if FABHALTA is discontinued and that they should be monitored by their healthcare providers for at least 2 weeks following discontinuation of FABHALTA. Inform patients who discontinue FABHALTA to keep the Patient Safety Card with them for 2 weeks after the last dose of FABHALTA. The increased risk of serious infection may continue for a few weeks after the last dose of FABHALTA. Hyperlipidemia Inform patients that FABHALTA may increase their cholesterol and triglycerides and that monitoring of these parameters will be needed periodically during treatment. Distributed by: Novartis Pharmaceuticals Corporation East Hanover, New Jersey 07936 For more information, visit www.FABHALTA.com or call 1-888-669-6682. © Novartis T2026-23
Label text from the FDA structured product label by Novartis Pharmaceuticals Corporation (revised Jul 16, 2026). Long sections are shortened; the complete label is on DailyMed.
Active ingredients
- Iptacopan Hydrochloride in 1 product
Fabhalta NDC products (1)
| NDC | Strength & form | Labeler | Type |
|---|---|---|---|
| 0078-1189 | Iptacopan Hydrochloride 200 mg/1 Capsule | Novartis Pharmaceuticals Corporation | NDA |
Frequently asked questions
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…
What are the side effects of Fabhalta?
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,… See the full label for the complete list.
Who makes Fabhalta?
Fabhalta is listed by 1 labeler in the FDA NDC directory, including Novartis Pharmaceuticals Corporation.