Iptacopan

Generic name: iptacopan
Brand name: Fabhalta
Dosage form: oral capsule (200 mg)
Drug class: Selective immunosuppressants

Medically reviewed by Drugs.com on Jan 29, 2024. Written by Cerner Multum.

What is iptacopan?

Iptacopan is used in adults to treat a disease called paroxysmal nocturnal hemoglobinuria (PNH).

Iptacopan is available only under a special program and from a certified pharmacy. You must understand the risks and benefits of iptacopan.

Iptacopan may also be used for purposes not listed in this medication guide.

Iptacopan side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

You may get infections more easily, even serious or fatal infections. Call your doctor right away if you have signs of infection such as:

• fever, chills, sore throat;

• mouth sores, red or swollen gums;

• pale skin, easy bruising, unusual bleeding;

• chest discomfort, wheezing, dry cough or hack, rapid weight loss; or

• symptoms of meningitis--headache, neck stiffness, increased sensitivity to light, nausea, vomiting, confusion, drowsiness.

Your condition may get worse if you stop taking this medicine. If you stop using iptacopan, tell your doctor if you have any new or worsening symptoms, such as tiredness, confusion, stomach pain, trouble breathing or swallowing, blood in your urine, difficulty having an erection, chest pain, pain in one leg, or numbness or weakness on one side of the body.

Common side effects of iptacopan may include:

• headache;

• infections caused by bacteria or virus;

• cold symptoms such as stuffy nose, sneezing, sore throat;

• rash; or

• diarrhea, nausea, stomach pain.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Related/similar drugs

Tarpeyo, Ultomiris, Soliris, eculizumab, Empaveli, Fabhalta, ravulizumab

Warnings

You may get infections more easily, even serious or fatal infections. Call your doctor if you have flu symptoms, cough, night sweats, neck stiffness, confusion, or vision problems.

Before taking this medicine

Before you start using iptacopan, tell your doctor if you have any signs of infection such as fever, chills, cough, body aches, tiredness, open sores or skin wounds, diarrhea, stomach pain, weight loss, painful urination, or coughing up blood.

You should not use iptacopan if you are allergic to it, or if you have:

• a serious infection caused by certain types of bacteria such as meningitis, pneumonia, or a bloodstream infection.

Tell your doctor if you have or have ever had:

• a chronic or recent infection;

• fever;

• high cholesterol or triglycerides;

• recently received a vaccine or are scheduled to receive a vaccine; or

• liver or kidney disease.

Make sure you are current on all vaccines that protect against serious bacterial infections at least 2 weeks before you start using iptacopan. If you were vaccinated in the past, you may need additional vaccinations or booster doses.

It is not known if iptacopan will harm an unborn baby. However, having paroxysmal nocturnal hemoglobinuria (PNH) during pregnancy may cause complications or medical problems in both mother and baby. The benefit of treating this condition may outweigh any risks to the baby.

Do not breastfeed while using iptacopan, and for at least 5 days after your last dose.

How should I take iptacopan?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

Read the Patient Wallet Card about serious side effects and learn what symptoms to watch for. Keep the card with you at all times, and for at least 2 weeks after your last dose.

If you are switching from another medicine such as eculizumab or ravulizumab, your first dose of iptacopan may be delayed. Follow your doctor's dosing instructions very carefully.

Iptacopan is usually taken twice a day, with or without food.

Swallow the capsule whole and do not crush, chew, break, or open it.

You may be given other medications to help treat or prevent a serious infection or certain side effects. Keep taking these medicines for as long as your doctor has prescribed.

You may get infections more easily, even serious or fatal infections. You will need frequent medical tests.

During your treatment, you will need frequent medical checkups and blood tests to check your cholesterol and triglycerides.

Your condition may get worse if you change your dose or stop using iptacopan suddenly. Ask your doctor before stopping the medicine. Your doctor will closely watch you for 2 weeks if you stop taking iptacopan.

Store at room temperature away from moisture and heat.

Iptacopan dosing information

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

What happens if I miss a dose?

Use the medicine as soon as you remember, and then go back to your regular schedule. Do not use two doses at one time.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking iptacopan?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect iptacopan?

Sometimes it is not safe to use certain medicines at the same time. Some drugs can affect your blood levels of other drugs you use, which may increase side effects or make the medicines less effective.

Other drugs may affect iptacopan, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

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

Medical Disclaimer

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