Fabhalta

Pronunciation: fab hal’ tah
Generic name: iptacopan
Dosage form: capsules (200mg)
Drug class: Selective immunosuppressants

Medically reviewed by Melisa Puckey, BPharm. Last updated on Dec 7, 2023.

What is Fabhalta?

Fabhalta (iptacopan) is a complement Factor B inhibitor used to treat paroxysmal nocturnal hemoglobinuria (PNH) in adults, to reduce anemia and the need for blood transfusions.  Fabhalta works to increase hemoglobin-levels by acting in the alternative complement pathway to help control the destruction of red blood cell (RBC) within and outside the blood vessels. 

Fabhalta received FDA approval on December 6, 2023, as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria. Fabhalta’s mechanism of action is it binds to Factor B of the alternative complement pathway which  prevents both intravascular (in the blood vessels) hemolysis and extravascular (mainly in liver in spleen) hemolysis.

Fabhalta capsules are taken twice daily with or without food.

What is Paroxysmal nocturnal hemoglobinuria?

Paroxysmal nocturnal hemoglobinuria is caused by an acquired genetic flaw that makes red blood cells that more likely to be destroyed early by the complement system. The red blood cells are destroyed within the blood vessels, the liver, and also the spleen, which causes anemia, formation of blood clots (thrombosis), and other symptoms. The classic symptom of PNH is dark red/brown urine that is more noticeable late at night or early in the morning; this is caused by the breakdown of RBC. 

Fabhalta side effects

The most common Fabhalta side effects were headache, diarrhea, abdominal pain, bacterial infection, viral infection, cold-like symptoms, nausea, and rash; these affected 10% of patients or more.

Serious Fabhalta side effects See “Warnings” section. 

Tell your healthcare provider about any side effect that bothers you or that does not go away. These are not all of the possible side effects of this medicine. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. 

Warnings

Fabhalta affects part of your immune system, it may lower the ability of your immune system to fight infections.

Fabhalta increases your chance of getting serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. These serious infections may quickly become life-threatening or fatal if not recognized and treated early. 

• You must complete or update your vaccinations against these bacteria at least 2 weeks before your first dose of Fabhalta.
• If you have not completed your vaccinations and Fabhalta must be started right away, you should receive the required vaccinations as soon as possible.
• If you have not been vaccinated and Fabhalta must be started right away, you should also receive antibiotics to take for as long as your healthcare provider tells you. 
• If you have been vaccinated against these bacteria in the past, you might need additional vaccinations before starting this medicine. Your healthcare provider will decide if you need additional vaccinations.
• Vaccines do not prevent all infections caused by encapsulated bacteria. Call your healthcare provider or get emergency medical care right away if you have any of these signs and symptoms of a serious infection:  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 fever, headache with stiff neck or stiff back, confusion, body aches with flu-like symptoms, clammy skin, eyes sensitive to light.

Your healthcare provider will give you a Patient Safety Card about the risk of serious infections. Carry it with you at all times during treatment and for 2 weeks after your last dose of this medicine. Your risk of serious infections may continue for a few weeks after your last dose of this medicine. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

Fabhalta is only available through the Fabhalta Risk Evaluation and Mitigation Strategy (REMS) program. Before you can take this medicine, your healthcare provider must:

• enroll in the Fabhalta REMS program
• counsel you about the risk of serious infections caused by certain bacteria
• give you information about the symptoms of serious infections
• make sure that you are vaccinated against serious infections caused by encapsulated bacteria and that you receive antibiotics if you need to start this medicine right away and you are not up to date on your vaccinations
• give you a Patient Safety Card about your risk of serious infections, as discussed above.

 This medicine may increase your cholesterol and triglycerides and your healthcare provider will do blood tests to check them periodically during treatment. 

Before taking this medicine

Before taking Fabhalta tell your healthcare provider about all of your medical conditions, including if you: 

• have an infection or fever
• have kidney or liver problems

You should not take this medicine if you are allergic to iptacopan or any of the inactive ingredients in Fabhalta. See the end of this page for a complete list of ingredients.

You should not take this medicine if you have a serious infection caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae type B.

Pregnancy

Tell your healthcare provider if you are pregnant or plan to become pregnant, as it is not known if Fabhalta will harm your unborn baby.

Breastfeeding

Tell your healthcare professional if you are breastfeeding or plan to breastfeed, as it is not known if Fabhalta passes into your breast milk. You should not breastfeed during treatment and for 5 days after your last dose of Fabhalta. 

How should I take Fabhalta?

Take Fabhalta exactly as your healthcare provider tells you. Do not change the dose or stop taking this medicine unless your healthcare provider tells you. 

Fabhalta Instructions

Take 1 capsule 2 times each day, with or without food. 

Swallow the capsules whole; do not open, break, or chew capsules.

Changing treatment from eculizumab to Fabhalta. You should take your starting dose of Fabhalta no later than 1 week after your last dose of eculizumab. 

Changing treatment from ravulizumab to Fabhalta.  You should take your starting dose of Fabhalta no later than 6 weeks after your last dose of ravulizumab. 

If you have PNH and you stop taking this medicine, your healthcare provider will need to monitor you closely for at least 2 weeks after stopping the capsules. Stopping treatment with Fabhalta may cause a breakdown of red blood cells due to PNH. Symptoms or problems that can happen due to breakdown of red blood cells include: 

• decreased hemoglobin level in your blood
• tiredness o blood in your urine
• pain in the stomach (abdomen)
• shortness of breath
• blood clots, stroke, and heart attack
• trouble swallowing
• erectile dysfunction.

 It is important that you take this medicine exactly as your healthcare provider tells you, to lower the possibility of breakdown of red blood cells due to PNH. 

What happens if I miss a dose?

If you miss a dose or doses of Fabhalta, take 1 dose as soon as you remember, even if it is almost time to take your next scheduled dose, and then take your next dose at your regularly scheduled time. 

Interactions

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking Fabhalta with certain other medicines may affect the way Fabhalta works and may cause side effects. Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. 

Storage

Store at room temperature between 68°F to 77°F (20°C to 25°C) in a container with a child-resistant cap. Ingredients

Ingredients 

Active ingredient: iptacopan

Inactive ingredients: the capsule shell contains 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. 

Company

Distributed by: Novartis Pharmaceuticals Corporation, East Hanover, New Jersey 07936.

Further information

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

Medical Disclaimer