Fabhalta is a brand-name oral capsule that’s prescribed for paroxysmal nocturnal hemoglobinuria. It’s also used for a certain type of kidney disease. Fabhalta contains the active ingredient iptacopan.

Fabhalta is FDA-approved to:

Drug details

You’ll find key information about Fabhalta below.

  • Drug class: complement inhibitor
  • Drug form: oral capsule
  • Generic available? no
  • Prescription required? yes
  • Controlled substance? no
  • Year of FDA approval: 2023
  • Accelerated approval? yes (for reducing proteinuria in certain adults with IgA nephropathy)

Fabhalta has a boxed warning. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). For details, see the “Fabhalta precautions” section.

Fabhalta is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics usually cost less than brand-name drugs.

The following information describes dosages that are commonly used or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your needs.

Drug forms and strengths

Fabhalta comes as an oral capsule. It’s available in one strength of 200 milligrams (mg).

Dosage for paroxysmal nocturnal hemoglobinuria

The recommended dosage of Fabhalta for paroxysmal nocturnal hemoglobinuria (PNH) in adults is 200 mg twice per day.

If you’re switching from a different PNH medication to Fabhalta, your doctor will advise you on how and when to switch. For example, if you’re switching from eculizumab (Soliris, Bkemv, Epysqli, others) to Fabhalta, your doctor will likely recommend starting Fabhalta within 1 week after your last dose of eculizumab. And if you’re switching from ravulizumab (Ultomiris), your doctor will likely recommend starting Fabhalta within 6 weeks after your last dose of ravulizumab.

Dosage for primary immunoglobulin A nephropathy

The recommended dosage of Fabhalta in adults with primary immunoglobulin A nephropathy (IgAN) is 200 mg twice per day.

About taking Fabhalta

Below you’ll find information about key dosage issues.

  • When to take: You should take Fabhalta twice per day. The typical schedule is one dose in the morning and one dose in the evening. Taking the medication around the same times each day helps keep a steady level of the drug in your body. This helps Fabhalta work effectively.
  • If you miss a dose: If you miss a dose or doses of Fabhalta, take one dose as soon as possible. You can do this even if your next scheduled dose is coming up soon. Then continue taking your usual dose on your regular schedule.
  • Taking Fabhalta with or without food: You can take Fabhalta with or without food. The timing of food doesn’t affect this medication.
  • Crushing, splitting, or chewing Fabhalta: Fabhalta comes as an oral capsule. You should not open, chew, or break the capsules. You should swallow Fabhalta whole.
  • Length of use: Fabhalta is meant to be used as a long-term treatment. If you and your doctor determine that Fabhalta is safe and effective for you, you’ll likely take it long term.
  • Length of time to work: Fabhalta starts working within 2 hours. But it may take several weeks to months to see the desired effect for treating your condition. Your doctor can tell you about when and how they’ll monitor you to check how well the medication is working.

Overdose

Do not take more Fabhalta than your doctor recommends. For some drugs, doing so may lead to unwanted side effects or overdose.

What to do in case you take too much Fabhalta

If you think you’ve taken too much of this drug, call your doctor. You can also call America’s Poison Centers at 800-222-1222 or use its online tool. However, if your symptoms are severe, call 911 or your local emergency number, or go to the nearest emergency room right away.

Fabhalta can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking Fabhalta. These lists do not include all possible side effects.

For more information about the possible side effects of Fabhalta, talk with your doctor or pharmacist. They can give you tips on how to manage any side effects that may be concerning or bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. If you would like to notify the FDA about a side effect you’ve had with Fabhalta, you can do so through MedWatch.

Mild side effects

Below is a partial list of mild side effects of Fabhalta. To learn about other mild side effects, talk with your doctor or pharmacist, or view Fabhalta’s prescribing information.

Mild side effects of Fabhalta can include:

Most of these side effects may go away within a few days to a couple of weeks. However, if they become more severe or don’t go away, talk with your doctor or pharmacist.

Serious side effects

Serious side effects from Fabhalta aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or you think you’re having a medical emergency.

Serious side effects and their symptoms can include:

* Fabhalta has a boxed warning for this side effect. This is the most serious warning from the FDA. To learn more, see the “Fabhalta precautions” section.

ALLERGIC REACTION

For some people, Fabhalta can cause an allergic reaction.

In general, symptoms of allergic reaction can be mild or serious.

Ways to manage

For mild allergic reaction symptoms, such as a mild rash, call your doctor right away. They may recommend treatments to help manage your symptoms. They’ll also let you know whether you should keep taking the medication.

For severe allergic reaction symptoms, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms require immediate medical care because they can become life threatening. If you’ve had a serious allergic reaction to Fabhalta oral capsule, your doctor may recommend taking a different medication instead.

The Food and Drug Administration (FDA) approves prescription drugs such as Fabhalta to treat certain conditions. Fabhalta may also be used off-label for other conditions. Off-label drug use is when an FDA-approved drug is prescribed for a purpose other than what it’s approved for.

Fabhalta for paroxysmal nocturnal hemoglobinuria

Fabhalta is FDA-approved to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) in adults.

With PNH, the immune system attacks the red blood cells in the body. This causes hemolysis, which means the red blood cells rupture (break apart). Red blood cells are important for carrying hemoglobin, a protein that carries oxygen throughout your body. Some people with PNH have mild symptoms (if any), such as fatigue. Others have severe symptoms, such as shortness of breath or blood clots, and may require regular blood transfusions.

In clinical studies, Fabhalta was shown to be an effective treatment for PNH based on a meaningful increase in hemoglobin levels, maintained hemoglobin levels, and a reduced need for blood transfusions.

Fabhalta for reducing proteinuria in immunoglobulin A nephropathy

The FDA also has granted an accelerated approval for Fabhalta for use in adults with a rare type of chronic kidney disease called primary immunoglobulin A (IgA) nephropathy, also known as IgAN or Berger’s disease. IgA is a blood protein that’s part of the immune system.

With IgAN, the IgA antibody builds up in the kidneys, leading to inflammation and increased amounts of protein in the urine. Higher amounts of protein in the urine can be a sign of declining kidney function. Over time, IgAN may lead to kidney failure, which is also known as end stage renal disease.

More specifically, Fabhalta is prescribed to reduce proteinuria (protein in the urine) in people who are considered at high risk for rapid disease progression (worsening). This is based on the ratio of protein and creatinine in the urine.

Accelerated approval means that the drug has not yet received full approval. Clinical studies for Fabhalta are still ongoing. It isn’t yet known whether the drug helps to slow disease progression in people with IgAN. The FDA’s decision to grant full approval is pending the results of ongoing clinical studies.

Fabhalta and children

Fabhalta is not approved for any uses in children.

Fabhalta can interact with several other medications.

Before taking Fabhalta, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you use. Sharing this information can help you avoid potential interactions.

Interactions with medications, foods, and supplements

Below is a list of medications, supplements, and foods that can interact with Fabhalta. This list does not contain all drugs that may interact with Fabhalta. If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

Medications that can interact with Fabhalta• gemfibrozil (Lopid)
• rifampin (Rifadin)

Alcohol interaction

It isn’t known whether alcohol interacts with Fabhalta.

If you have questions about how much alcohol is safe to drink while taking Fabhalta, talk with your doctor or pharmacist.

As with all medications, the cost of Fabhalta can vary. The actual price you’ll pay depends on your insurance plan, your location, and the pharmacy you use.

Financial and insurance assistance: If you need financial support to pay for Fabhalta, or if you need help understanding your insurance coverage, help is available.

A program called Novartis Patient Support is available for Fabhalta. For more information and to find out if you’re eligible for support, call 833-993-2242 or visit the program website.

Generic version:Fabhalta is not available in a generic form. A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics tend to cost less than brand-name drugs.

To learn more about saving money on prescriptions, check out this article.

Other drugs are available that can treat your condition. Some may be a better fit for you than others. If you’re interested in finding an alternative to Fabhalta, talk with your doctor. They can tell you about other medications that may work well for you.

The following drugs are similar to Fabhalta:

  • pegcetacoplan (Empaveli)
  • danicopan (Voydeya)

If you can become pregnant, consider the following information about pregnancy, birth control, and breastfeeding.

Fabhalta and pregnancy

It’s not known whether Fabhalta should be taken during pregnancy. If you’re planning a pregnancy or can become pregnant, talk with your doctor before taking this medication.

Fabhalta and birth control

It’s not known if Fabhalta is safe to take during pregnancy. If you or your partner can become pregnant, talk with your doctor about your birth control needs while taking Fabhalta.

Fabhalta and breastfeeding

It’s not known whether Fabhalta should be taken while breastfeeding. Tell your doctor if you’re currently breastfeeding or planning to do so. Due to the potential for side effects in a breastfed child, your doctor will likely recommend other ways to feed your child during Fabhalta treatment and for at least 5 days after your last dose of Fabhalta.

Here are answers to some frequently asked questions about Fabhalta.

When did Fabhalta receive FDA approval?

The Food and Drug Administration (FDA) first approved Fabhalta in December 2023. At that time, Fabhalta had only one indication (approved use) to treat paroxysmal nocturnal hemoglobinuria (PNH) in adults.

In August 2024, the FDA granted an accelerated approval for Fabhalta for another use to reduce proteinuria (protein in the urine) in certain adults with primary immunoglobulin A nephropathy (IgAN). IgAN is a type of chronic kidney disease.

Accelerated approval means that the drug has not yet received full approval. Clinical studies for Fabhalta are still ongoing. It isn’t yet known whether the drug helps slow disease progression in people with IgAN. The FDA’s decision to grant full approval is pending the results of ongoing clinical studies.

For more information about Fabhalta’s indications, see the Uses section.

What are the risks of stopping Fabhalta for PNH?

Stopping Fabhalta may cause the symptoms of PNH to worsen or come back, including hemolysis (rupture of red blood cells). Symptoms of hemolysis may include:

Hemolysis may also increase the risk of serious cardiovascular problems, such as blood clots, stroke, or heart attack.

Because of these risks, it’s important not to stop taking Fabhalta unless your doctor tells you to. If they instruct you to stop Fabhalta, they’ll likely prescribe a different medication to treat your condition.

It’s also important not to miss doses of Fabhalta. Fabhalta is a short-acting medication and requires twice-daily dosing to keep working effectively.

Talk with your doctor or pharmacist if you have questions about stopping Fabhalta.

How does Fabhalta work?

Fabhalta works by targeting a part of your immune system known as the complement pathway.

With PNH, your immune system attacks red blood cells, causing them to rupture (break apart). Fabhalta treats PNH by blocking specific steps in the complement pathway. This helps stop or reduce the breakdown of red blood cells.

With IgAN, the antibody immunoglobulin A builds up in your kidneys, causing inflammation and increased protein in the urine. Fabhalta works for IgAN by blocking specific actions in the complement pathway. This helps reduce the amount of protein in the urine.

If you have questions about how Fabhalta works or what results to expect during treatment, talk with your doctor.

This drug comes with several precautions.

FDA warnings

This drug has a boxed warning for the risk of serious bacterial infection. A boxed warning is the most serious warning from the Food and Drug Administration (FDA). It alerts doctors and patients about drug effects that may be dangerous.

Fabhalta may increase the risk of serious bacterial infections, especially a type called encapsulated bacteria. Examples of serious infections caused by encapsulated bacteria are pneumonia, meningitis, and bloodstream infections. If not caught and treated quickly, these infections can be fatal.

To reduce the risk of these infections, you should ideally be up to date on all routine vaccinations before you start Fabhalta, especially vaccinations that help prevent infections caused by encapsulated bacteria. Your doctor can recommend whether you need any vaccines or booster doses before you start Fabhalta.

If you’re not currently up-to-date on these vaccines, you should ideally get them at least 2 weeks before your first dose of Fabhalta. This allows enough time for the vaccines to work. If your doctor wants you to start Fabhalta sooner than this, they’ll likely give you antibiotics to take for the first 2 weeks of Fabhalta treatment. This is to help protect against infections until the vaccines start working.

Vaccination reduces the risk of these serious bacterial infections. Still, it’s important to contact your doctor right away if you have symptoms of an infection, such as:

  • headache with nausea and vomiting
  • stiff neck or back
  • fever with or without skin rash
  • rapid heart rate
  • clammy skin
  • confusion
  • flu-like symptoms, such as cough, congestion, body aches, and chills

Due to this risk, Fabhalta is only available through a special program called the Fabhalta Risk Evaluation and Mitigation Strategy (REMS). Doctors must enroll in this program before they can prescribe Fabhalta, and only certified pharmacies can dispense it. Your doctor will talk with you about the importance of vaccinations, taking any prescribed antibiotics as directed, and recognizing early signs of infections. You’ll also receive a Patient Safety Card, which you should carry at all times during Fabhalta treatment and for 2 weeks after your last dose.

Other precautions

Before taking Fabhalta, discuss your health history with your doctor. Fabhalta may not be right for you if you have certain medical conditions or other factors affecting your health. Be sure to talk with your doctor if any of the following apply to you:

  • severe liver problems
  • infection or fever
  • previous allergic reaction to this or a similar drug
  • pregnancy
  • breastfeeding

Note: For more information about the potential negative effects of Fabhalta, see the “Fabhalta side effects” section.

Disclaimer: Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.