Fabhalta

Pronunciation: fab-hal-tah
Generic name: iptacopan
Dosage form: oral capsule (200 mg)
Drug class: Selective immunosuppressants

Medically reviewed by Carmen Pope, BPharm. Last updated on Mar 26, 2025.

What is Fabhalta?

Fabhalta is used to treat paroxysmal nocturnal hemoglobinuria (PNH), primary immunoglobulin A nephropathy (IgAN), or complement 3 glomerulopathy (C3G) in adults. One capsule is taken twice a day.

Fabhalta is only available through the Fabhalta REMS (Risk Evaluation and Mitigation Strategy) program. Before you can take Fabhalta, your healthcare provider must counsel you about infection risks, provide a patient safety card, ensure proper vaccinations, and give antibiotics if needed before starting treatment.

Fabhalta (iptacopan) first gained FDA approval on December 5, 2023. There is no generic.

FDA-approved uses and indications

Fabhalta is approved to treat adults with:

• paroxysmal nocturnal hemoglobinuria (PNH)
• primary immunoglobulin A nephropathy (IgAN) to reduce proteinuria (protein in the urine) in those at risk of their disease progressing quickly (generally a urine protein-to-creatinine ratio [UPCR] of ≥ 1.5 g/g)
  • This approval is under the accelerated approval designation, and continued approval may be contingent upon a clinical benefit being shown in clinical trials.
• complement 3 glomerulopathy (C3G), to reduce levels of proteinuria.

It is not known if Fabhalta is safe and effective in children with PNH, IgAN, or C3G.

How does Fabhalta work?

Fabhalta's mechanism of action involves blocking a protein called complement factor B, which plays a key role in the breakdown of red blood cells. Taking Fabhalta prevents RBC destruction, anemia, and proteinuria, and reduces kidney damage.

Side effects

The most common side effects of Fabhalta are:

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

Serious side effects and warnings

Fabhalta carries a Boxed Warning for serious infections caused by encapsulated bacteria. It may also cause the following severe side effects:

An increased risk of serious infections. 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
  • sensitivity 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.

Hemolysis. 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.

Increase cholesterol and triglyceride levels.  Your healthcare provider will do blood tests regularly to check your lipid levels 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.

Related/similar drugs

Before taking

Do not take Fabhalta if you:

• are allergic to Fabhalta, iptacopan, or any ingredients in the capsules.
• 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.

• 1 capsule is taken 2 times a day.
• Swallow the capsules whole. Do not open, break, or chew them.
• May be taken with or without food.

Stopping Fabhalta when you have PNH

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

Take Fabhalta exactly as your healthcare provider tells you to lower the possibility of breakdown of red blood cells due to PNH.

Dosing information

Dosage for PNH, IgAN, or C3G: Fabhalta 200 mg (1 capsule) twice a day.

See the Fabhalta Prescribing Information for more detailed dosing information.

What happens if I miss a dose?

If you miss a dose or doses of Fabhalta, take one 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 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 it 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 at room temperature, in the original container.

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

Ingredients

Active ingredient: iptacopan 

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.

Available as 200 mg capsules.

Manufacturer

Novartis Pharmaceuticals Corporation makes Fabhalta.

Further information

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

Medical Disclaimer