Brand Name
Fabhalta
Generic Name
Iptacopan
View Brand Information FDA approval date: December 05, 2023
Classification: Complement Factor B Inhibitor
Form: Capsule
What is Fabhalta (Iptacopan)?
FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria .
Approved To Treat
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Brand Information
FABHALTA (iptacopan)
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
- 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
- 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
1DOSAGE 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.
2CONTRAINDICATIONS
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
3ADVERSE REACTIONS
The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling:
- Serious Infections Caused by Encapsulated Bacteria
- Hyperlipidemia
3.1Clinical 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.
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
The data below reflect FABHALTA exposure in 235 patients with IgAN (eGFR ≥ 20 mL/min/1.73 m
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 (
4DESCRIPTION
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
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.
5HOW SUPPLIED/STORAGE AND HANDLING
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].
6PATIENT COUNSELING 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
Inform patients that vaccination may not prevent serious infection and to seek immediate medical attention if the following signs or symptoms occur
- 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
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.
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© Novartis
T2025-15
7PRINCIPAL DISPLAY PANEL
NDC 0078-1189-20
Rx only
FABHALTA
200 mg
Dispense with accompanying Medication Guide.
60 capsules
Swallow the capsules whole. Do not open, break, or chew capsules.
NOVARTIS
