Iptacopan Dosage

Medically reviewed by Drugs.com. Last updated on Jan 29, 2024.

Usual Adult Dose for Paroxysmal Nocturnal Hemoglobinuria

200 mg orally twice a day

Comments:

• Administer vaccinations against encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis (serogroups A, C, W, Y, and B), and Haemophilus influenzae type B, following current Advisory Committee on Immunization Practices (ACIP) recommendations, at least 2 weeks before initiating this drug.
• In cases where urgent treatment with this drug is necessary for a patient who does not have up-to-date vaccinations for S pneumoniae, N meningitidis, and H influenzae type B as per ACIP guidelines, provide antibacterial drug prophylaxis and promptly administer the required vaccines.
• To minimize the potential hemolysis risk associated with the sudden cessation of alternative paroxysmal nocturnal hemoglobinuria (PNH) therapies:
• For patients transitioning from eculizumab, commence treatment with this drug within 1 week following the final eculizumab dose.
• For those transitioning from ravulizumab, initiate treatment with this drug no later than 6 weeks after the last ravulizumab dose.
• Information on the recommended timeframe for starting this drug after discontinuing other PNH therapies is currently unavailable.

Use: Treatment of patients with PNH

Renal Dose Adjustments

Mild to moderate renal dysfunction (estimated glomerular filtration rate [eGFR] 30 to less than 90 mL/min/1.73 m2): No adjustment recommended
Severe renal dysfunction (eGFR less than 30 mL/min/1.73 m2): Not recommended

Liver Dose Adjustments

Mild or moderate liver dysfunction (Child-Pugh A or B): No adjustment recommended
Severe liver dysfunction (Child-Pugh C): Not recommended

Precautions

The US FDA requires a Risk Evaluation and Mitigation Strategy (REMS) for iptacopan. It includes elements to assure safe use and an implementation system. For additional information: www.accessdata.fda.gov/scripts/cder/rems/index.cfm

US BOXED WARNING:

• SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA: This drug increases the likelihood of serious infections, especially arising from encapsulated bacteria (such as S pneumoniae, N meningitidis, and H influenzae type B). Life-threatening and fatal infections with encapsulated bacteria have occurred with complement inhibitors; such infections may become rapidly life-threatening or fatal if not recognized and treated early. Ensure completion or up-to-date status of vaccinations targeting encapsulated bacteria at least 2 weeks before the initial dose of this drug, unless the risks of delaying the medication outweigh the risk of developing a serious infection. Adhere to the latest ACIP recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor; the manufacturer product information should be consulted for further guidance managing risk of serious infections. Patients using this drug face an increased susceptibility to invasive disease caused by encapsulated bacteria, even if they generate antibodies postvaccination. Monitor patients closely for early indications and symptoms of serious infections, and promptly assess if infection is suspected. Due to the risk of serious infections caused by encapsulated bacteria, this drug is available only through a restricted program under a REMS.

CONTRAINDICATIONS:

• Serious hypersensitivity to the active component or any of the ingredients
• Initiation with unresolved serious infection caused by encapsulated bacteria, including S pneumoniae, N meningitidis, or H influenzae type B

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Dialysis

Hemodialysis: Not recommended

Other Comments

Administration advice:

• This drug can be administered with or without food.
• Swallow capsules whole. Do not open, break, or chew capsules.
• In case of a missed dose, take 1 dose of this drug as soon as possible (even if it is soon before the next scheduled dose) and then resume the regular dosing schedule.

Storage requirements:

• Store at 20C to 25C (68F to 77F); excursions permitted between 15C and 30C (59F and 86F).

General:

• This drug is available only through a restricted program under a REMS called FABHALTA REMS.

Monitoring:

• Infections/Infestations: For early signs and symptoms of serious infections
• Hematologic: For signs of hemolysis after discontinuation
• Metabolic: Serum lipid parameters periodically during treatment

Patient advice:

• Read the US FDA-approved patient labeling (Medication Guide).
• 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 influenza-like symptoms
• clammy skin
• eyes sensitive to light
• Always carry Patient Safety Card for this drug during and for 2 weeks following treatment with this drug.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer