Iptacopan

Iptacopan is a prescription medication indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 

• Iptacopan is available under the following different brand names: Fabhalta.

Common side effects of Iptacopan include:

• headache 
• pain in the stomach (abdomen)
• nasal congestion, runny nose, cough, sneezing, and sore throat (nasopharyngitis)
• infections (viral and bacterial)
• nausea
• diarrhea 
• rash
• Serious side effects of Iptacopan include:
• infections
• fever with or without shivers or chills
• rash
• chest pain 
• cough 
• breathlessness/fast breathing
• high heart rate 
• headache 
• nausea or vomiting
• stiff neck or stiff back
• confusion 
• body aches with flu-like symptoms
• clammy skin 
• eyes sensitive to light
• increased total cholesterol, LDL-cholesterol, and serum triglyceride levels

Rare side effects of Iptacopan include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Paroxysmal nocturnal hemoglobinuria

• 200 mg orally two times a day
• Switching from complement C5 inhibitors to Iptacopan
• Reduce the potential risk of hemolysis with abrupt discontinuation of other PNH therapies
• Switching from eculizumab: Initiate Iptacopan no later than 1 week after the last eculizumab dose
• Switching from ravulizumab: Initiate Iptacopan no later than 6 weeks after last ravulizumab dose
• Information is unavailable regarding the timeframe for initiating Iptacopan after other PNH therapies

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Iptacopan has severe interactions with no other drugs
• Iptacopan has serious interactions with the following drug:
  • gemfibrozil
• Iptacopan has moderate interactions with the following drugs:
  • carbamazepine
  • fosphenytoin
  • mitapivat
  • nilotinib
  • phenobarbital
  • phenytoin
  • primidone
  • resmetirom
  • rifampin
  • secobarbital
• Iptacopan has minor interactions with no other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• Serious hypersensitivity to Iptacopan or any of the excipients
• Unresolved serious infection with encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type B

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Iptacopan?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Iptacopan?”

Cautions

• Monitor PNH manifestations after discontinuing Iptacopan
• After discontinuing, closely monitor for at least 2 weeks after the last dose for signs and symptoms of hemolysis
• 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 (eg, thrombosis, stroke, and myocardial infarction), dysphagia, or erectile dysfunction
• If discontinuation is necessary, consider alternative therapy
• If hemolysis occurs after discontinuation, consider restarting Iptacopan, if appropriate, or initiating another treatment for PNH
• Hyperlipidemia
  • Iptacopan may increase total cholesterol, LDL-cholesterol, and serum triglycerides
  • May require cholesterol-lowering medications
  • Monitor serum lipid parameters periodically during treatment and initiate cholesterol-lowering medication, if indicated
• Serious infections caused by encapsulated bacteria
  • Increased 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
  • Contraindicated in patients with unresolved serious infections caused by encapsulated bacteria
• Complete or update vaccination against encapsulated bacteria at least 2 weeks before the first dose, according to current ACIP recommendations for patients receiving a complement inhibitor
  • Revaccinate patients by ACIP recommendations considering the duration of therapy with Iptacopan
  • Note that ACIP recommends an administration schedule in patients receiving complement inhibitors that differs from the standard administration schedule
  • If urgent therapy is indicated in a patient who is not current with vaccines against encapsulated bacteria, provide antibacterial drug prophylaxis and administer these vaccines as soon as possible
  • Vaccination does not eliminate the risk of serious encapsulated bacterial infections, despite the development of antibodies following vaccination
  • Closely monitor for early signs and symptoms of serious infection and evaluate patients immediately if an infection is suspected
  • Consider interruption of therapy in patients who are undergoing treatment for serious infections; serious infections may become rapidly life-threatening or fatal if not recognized and treated early
• REMS
  • 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 vaccination status for vaccines against encapsulated bacteria and vaccinate if needed according to current ACIP recommendations 2 weeks before the first dose of Iptacopan
  • Prescribers must provide a prescription for antibacterial drug prophylaxis if treatment must be started urgently in the patient who is not current with vaccines against encapsulated bacteria according to current ACIP recommendations at least 2 weeks before the first Iptacopan dose
  • Pharmacies that dispense Iptacopan 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 always carry the Patient Safety Card with them during treatment and for 2 weeks following the last dose
  • Further information is available by telephone: at 1-833-99FABHA or online at www.fabhalta-rems.com
• Drug interaction overview
  • CYP2C8 substrate (major)
  • CYP2C8 inducers
  • Monitor
    • Coadministration may decrease Iptacopan exposure, which may result in loss of or reduced efficacy
    • Monitor clinical response and discontinue CYP2C8 inducer if loss of Iptacopan efficacy is evident
  • Strong CYP2C8 inhibitors
  • Avoid
    • Coadministration-strong CYP2C8 inhibitors may increase Iptacopan exposure, which may result in adverse reactions

Pregnancy and Lactation

• Data from clinical trials are insufficient regarding use in pregnant women to identify the drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes
• Use in pregnant women or those planning to become pregnant may be considered following an assessment of the risks and benefits
• Clinical considerations
  • 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
• Lactation
  • Data are unavailable on the presence of Iptacopan or its metabolite in either human or animal milk, its effects on breastfed children, or on milk production
  • Discontinue breastfeeding during treatment and for 5 days after the final dose