Fabhalta
Fabhalta (iptacopan) is a complement inhibitor used to treat adults with a rare blood condition called paroxysmal nocturnal hemoglobinuria (PNH). It also helps to reduce protein levels in the urine for certain people with a kidney condition called primary immunoglobulin A nephropathy (IgAN). This medication comes as a capsule that you take by mouth twice a day. Common side effects include headache, diarrhea, and stomach pain. Fabhalta (iptacopan) is only available through a special Risk Evaluation and Mitigation Strategies (REMS) program since it raises the risk for serious infections.
What is Fabhalta (iptacopan)?
What is Fabhalta (iptacopan) used for?
To reduce protein levels in the urine for adults with primary immunoglobulin A nephropathy (IgAN) at high risk for getting worse
How Fabhalta (iptacopan) works
Fabhalta (iptacopan) is a complement inhibitor. It works by blocking a part of the immune system called the alternative complement pathway. This helps control a chain of immune reactions that can lead to damage in certain conditions.
In PNH, the immune system attacks red blood cells leading to their destruction. Fabhalta (iptacopan) helps protect these cells by blocking the complement system early in the process, reducing symptoms of PNH.
In IgAN, certain immune proteins build up in the kidneys and cause inflammation, which can lead to kidney damage over time. By blocking the alternative complement pathway, Fabhalta (iptacopan) has been shown to reduce protein levels in the urine, potentially protecting the kidney.
Drug Facts
What are the side effects of Fabhalta (iptacopan)?
Common Side Effects
- Headache (19%)
- Common cold symptoms (16%)
- Diarrhea (15%)
- Stomach pain (15%)
- Bacterial infection (11%)
- Nausea (10%)
- Viral infection (10%)
- Joint pain (8%)
- Upper respiratory tract infection (9%)
- High cholesterol/triglycerides (6%)
- Stomach pain (6%)
Other Side Effects
- Low platelet count
- Dizziness
- High blood pressure
- High cholesterol/triglycerides
- Rash
- Nausea
- Dizziness
Serious Side Effects
- Serious infections: fever, weakness, chills, trouble breathing, cough, diarrhea, stomach pain, burning when you urinate, urinating more or less often, neck pain, neck stiffness, confusion, severe headache
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Infection—fever, chills, cough, sore throat, wounds that don't heal, pain or trouble when passing urine, general feeling of discomfort or being unwell
- Fever, neck pain or stiffness, sensitivity to light, headache, nausea, vomiting, confusion, which may be signs of meningitis
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Diarrhea
- Headache
- Nausea
- Runny or stuffy nose
- Sore throat
- Stomach pain
Pros and cons of Fabhalta (iptacopan)
Pros
Works well to raise hemoglobin levels
Comes as a capsule that's taken by mouth
Can take with or without food
Cons
Raises your risk for serious infections
Only available through a special REMs program
Hasn't been studied in children
Pharmacist tips for Fabhalta (iptacopan)
Take Fabhalta (iptacopan) with or without food.
Swallow the Fabhalta (iptacopan) capsule whole. Don't open, break, or chew the capsules since this can affect how the medication works.
Take Fabhalta (iptacopan) as prescribed every day and try not to miss any doses. Missing doses can raise your risk of hemolysis, or red blood cell breakdown.
If you miss a dose of Fabhalta (iptacopan), take it as soon as you remember, even if it's close to your next scheduled dose. Then continue with your regular dosing schedule.
Since Fabhalta (iptacopan) raises your risk for serious bacterial infections, you'll need to receive vaccinations at least 2 weeks before starting the medication. If you need to start Fabhalta (iptacopan) right away, your prescriber will recommend antibiotics to help prevent infections.
Get medical help right away if you have symptoms of an infection while taking Fabhalta (iptacopan), such as fever, cough, trouble breathing, weakness, sweating, chills, or neck pain and stiffness. Vaccination lowers the risk but does not fully prevent bacterial infections.
Carry the Patient Safety Card with you at all times while taking Fabhalta (iptacopan) and for 2 weeks after stopping it. This is required as part of the REMS program for your safety.
Fabhalta (iptacopan) can raise your cholesterol and triglyceride levels. Your prescriber might monitor these levels with regular lab tests and recommend medication if needed to keep them in a healthy range.
Frequently asked questions about Fabhalta (iptacopan)
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What are the risks and warnings for Fabhalta (iptacopan)?
Fabhalta (iptacopan) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Serious infections
Risk factors: Current infection | Past infections that keep coming back | Other conditions that raise your risk of infection | Taking medications that weaken your immune system
Fabhalta (iptacopan) can weaken your body's ability to fight off infections, raising the risk for serious and life-threatening infections, like meningitis, pneumonia, and Haemophilus influenzae disease (Hib disease). Before starting Fabhalta (iptacopan), you must be up to date on vaccines. You might need the pneumonia vaccine, meningitis vaccine, and the Hib vaccine at least 2 weeks before your treatment begins. Your healthcare professional (HCP) will check if you need additional vaccines or preventive antibiotics before and during treatment. Even if you are vaccinated, you can still get serious infections. Get medical help right away if you have signs of an infection, such as fever, chills, body aches, cough, nausea, vomiting, eyes that are sensitive to light, neck pain or stiffness, severe headache, or confusion.
Because of the risk for serious infections, Fabhalta (iptacopan) is only available through a restricted Risk Evaluation and Mitigation Strategies (REMS) program called Fabhalta REMS. Your HCP must enroll in this program to prescribe it. They will also discuss infection risks with you and how to stay protected. During treatment and for 2 weeks after your last dose, you must carry a Patient Safety Card at all times in case of an emergency.
Return of PNH symptoms after stopping the medication
If you have PNH and stop taking Fabhalta (iptacopan), your prescriber will monitor you for at least 2 weeks for signs of hemolysis (red blood cell breakdown). Contact them if you notice tiredness, stomach pain, shortness of breath, blood in your urine, trouble swallowing, or erectile dysfunction. If signs of hemolysis appear after stopping, your prescriber might consider restarting Fabhalta (iptacopan) or switching you to a different PNH medication.
High cholesterol levels
Fabhalta (iptacopan) can raise your blood cholesterol and triglyceride levels. Your prescriber will likely order regular blood tests during treatment to make sure your levels stay in a safe range. If your cholesterol gets too high, they might prescribe a cholesterol-lowering medication or consider stopping Fabhalta (iptacopan).
Fabhalta (iptacopan) dosage forms
Typical dosing for Fabhalta (iptacopan)
The typical dose is 200 mg by mouth twice a day with or without food.
Fabhalta (iptacopan) contraindications
Current infection caused by the bacteria Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae type B
What are alternatives to Fabhalta (iptacopan)?
To reduce protein levels in the urine for adults with primary immunoglobulin A nephropathy (IgAN) at high risk for getting worse
Fabhalta (iptacopan) images
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References
Best studies we foundCenters for Disease Control and Prevention. (n.d.). Haemophilus influenzae disease.
de Latour, R. P., et al. (2024). Oral iptacopan monotherapy in paroxysmal nocturnal hemoglobinuria. The New England Journal of Medicine.
Janeway, C. A., Jr., et al. (2001). The complement system and innate immunity. Immunobiology: The Immune System in Health and Disease. 5th edition.
MedlinePlus. (2024). Hemolysis.
National Kidney Foundation. (2024). IgA nephropathy (IgAN).
Novartis AG. (2023). Novartis receives FDA approval for Fabhalta® (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH.
Novartis Pharmaceuticals Corporation. (2024). Fabhalta- iptacopan capsule [package insert]. DailyMed.
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