Fabhalta

Pronunciation: fab hal’ tah
Generic name: iptacopan
Dosage form: oral capsule
Drug class: Selective immunosuppressants

Medically reviewed by Carmen Pope, BPharm. Last updated on Aug 18, 2024.

What is Fabhalta?

Fabhalta (iptacopan) is an oral complement Factor B inhibitor that may be used to:

• treat adults with paroxysmal nocturnal hemoglobinuria (PNH)
• reduce protein levels in the urine (proteinuria) in adults with a kidney disease called primary immunoglobulin A nephropathy (IgAN), at risk of their disease progressing quickly.

Fabhalta targets a part of the immune system and blocks a protein called complement factor B, which plays a key role in the breakdown of red blood cells. Fabhalta's mechanism of action in PNH is to prevent RBC destruction, preventing anemia, and the need for blood transfusions. For IgAN, the complement system is thought to contribute to the kidney damage associated with IgAN, and Fabhalta helps manage this damage by specifically targeting and blocking complement factor B.

There is no Fabhalta generic available. Iptacopan is only available under the brand name Fabhalta.

Fabhalta gained FDA approval on December 5, 2023, for PNH. On August 8, 2024, approval was extended under the accelerated approval designation to include adults with IgAN at risk of disease progression. Continued approval for Fabhalta for IgAN is contingent upon a continued benefit being demonstrated in clinical trials.

Fabhalta REMS

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

• enroll you in the Fabhalta REMS program
• counsel you about the risk of serious infections caused by certain bacteria
• give you information and a patient safety card 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 Fabhalta immediately but are not up to date on your vaccinations
• give you a Patient Safety Card about your risk of serious infections.

Fabhalta side effects

The most common side effects of Fabhalta include:

• headache
• nasal congestion, runny nose, cough, sneezing, and sore throat (nasopharyngitis)
• diarrhea
• pain in the stomach (abdomen)
• infections (viral and bacterial)
• nausea
• rash.

Serious side effects and warnings

Fabhalta carries a Boxed Warning for serious infections caused by encapsulated bacteria.

Fabhalta may lower the ability of your immune system to fight infections and increase your risk of serious infections caused by encapsulated bacteria, such as 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 be up to date with the vaccines against S. pneumoniae and N. meningitidis 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 and 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.

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, chills, a rash, chest pain and cough, breathlessness/fast breathing, headache, or high heart rate)
• headache with nausea or vomiting or 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 Fabhalta. Your risk of serious infections may continue for a few weeks after your last dose of Fabhalta. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

There is a risk of hemolysis (red blood cell destruction) after stopping Fabhalta for PNH. Tell your healthcare provider immediately if you experience any of the following after you have stopped taking Fabhalta:

• Fatigue
• Weakness
• Dizziness
• Pale skin
• Jaundice (yellowing of the skin and eyes)
• Dark urine
• Rapid heart rate
• Shortness of breath
• Enlarged spleen.

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

It is not known if Fabhalta is safe and effective in children.

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

Before taking

Do not take Fabhalta if you:

• are allergic to Fabhalta or any of the ingredients in Fabhalta. See the end of this guide for a list of ingredients
• have a serious infection when starting treatment
• have severe kidney or liver disease.

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

• have an infection or fever
• have kidney or liver problems
• are not up to date with your vaccinations
• have an infection
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Pregnancy

It is not known if Fabhalta will harm your unborn baby.

Breastfeeding

It is not known if Fabhalta passes into your breast milk. Do not breastfeed during treatment and for 5 days after your last dose.

How should I take Fabhalta?

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

• The usual Fabhalta dosage is 200 mg (1 capsule), 2 times a day.
• Fabhalta may be taken with or without food.
• Swallow the capsules whole. Do not open, break, or chew capsules.

Changing from other PNH treatments

Your healthcare provider will give you instructions when changing from one PNH treatment to another. If you are changing treatment from:

• eculizumab (Soliris) to Fabhalta, you should take your starting dose of Fabhalta no later than 1 week after your last dose of eculizumab
• ravulizumab (Ultomiris) to Fabhalta, you should take your starting dose of Fabhalta no later than 6 weeks after your last dose of ravulizumab.

Fabhalta dosing information

Fabhalta dosage for PNH or IgAN: Fabhalta 200 mg 2 times a day with or without food.

Related/similar drugs

Empaveli, budesonide, cyclophosphamide, Cytoxan, eculizumab, Ultomiris, Soliris, ravulizumab, Filspari, Tarpeyo

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. Then take your next dose of Fabhalta at your regularly scheduled time.

What happens if I stop taking Fabhalta?

If you have PNH and you stop taking Fabhalta your healthcare provider will need to monitor you closely for at least 2 weeks after stopping, because stopping treatment may cause a breakdown of red blood cells (hemolysis) due to PNH.

Symptoms or problems that can happen due to the breakdown of red blood cells after stopping Fabhalta include:

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

What other drugs affect Fabhalta?

Tell your healthcare provider about all your medicines, 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. Especially tell your healthcare provider if you take:

• CYP2C8 inducers, such as rifampin. These may decrease the effectiveness of Fabhalta
• Strong CYP2C8 inhibitors, such as gemfibrozil. These may increase the blood levels of Fabhalta.

Know the medicines you take and the vaccines you receive. Keep a list to show your healthcare provider and pharmacist when you get a new medicine. See the Fabhalta Package Insert for a list of interactions.

Storage

Store Fabhalta capsules at room temperature between 68°F to 77°F (20°C to 25°C).

Keep out of the reach of children even though the Fabhalta container has a child-resistant cap.

Fabhalta ingredients

Active ingredient: iptacopan 200 mg

Inactive ingredients, capsule shell: gelatin, red ferric oxide, titanium dioxide, yellow ferric oxide.

Black printing ink: ferrosoferric oxide, potassium hydroxide, propylene glycol, shellac, and strong ammonia solution.

Who makes Fabhalta?

Novartis Pharmaceuticals Corporation manufactures Fabhalta.

Further information

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

Medical Disclaimer