FABHALTA (Iptacopan)
Oral Complement Factor B Inhibitor for PNH and C3G
FABHALTA (iptacopan) is the first oral monotherapy complement factor B inhibitor approved for treating paroxysmal nocturnal hemoglobinuria (PNH) in adults with hemolytic anemia, and complement factor 3 glomerulopathy (C3G). By selectively targeting factor B in the alternative complement pathway, FABHALTA prevents complement-mediated destruction of red blood cells and kidney damage. This page provides comprehensive, evidence-based information about FABHALTA including uses, dosage, side effects, and drug interactions.
Quick Facts
Key Takeaways
- FABHALTA is the first oral complement factor B inhibitor, offering a convenient oral alternative to intravenous complement therapies for PNH and C3G.
- The standard dose is 200 mg twice daily (morning and evening), taken as a capsule with or without food.
- Vaccination against meningococcal and pneumococcal bacteria is mandatory before starting treatment due to increased infection risk.
- Common side effects include upper respiratory infections, headache, and diarrhea; serious infections with encapsulated bacteria are the most significant risk.
- Treatment should not be stopped abruptly in PNH patients, as this can trigger hemolytic rebound with worsening anemia and potential blood clots.
What Is FABHALTA and What Is It Used For?
FABHALTA contains the active substance iptacopan, which belongs to a group of medicines called complement inhibitors. These drugs work by targeting specific components of the complement system, a part of the body's innate immune defense. In certain diseases, the complement system becomes overactive and attacks the body's own tissues, leading to serious health consequences.
FABHALTA is approved for two distinct medical conditions. The first is paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired blood disorder in which the immune system's complement system attacks and destroys red blood cells (a process called hemolysis). This destruction leads to anemia (low red blood cell count), fatigue, difficulty functioning, pain, dark-colored urine, shortness of breath, difficulty swallowing, erectile dysfunction, and an increased risk of blood clots (thrombosis). PNH affects approximately 1-1.5 per million people worldwide and can significantly impair quality of life.
The second approved indication is complement factor 3 glomerulopathy (C3G), a rare kidney disease in which overactive complement causes abnormal deposits of complement protein C3 in the glomeruli (the filtering units of the kidneys). This accumulation triggers inflammation and fibrosis (scarring), leading to high levels of protein in the urine (proteinuria) and progressive loss of kidney function. Without treatment, C3G can progress to end-stage kidney disease requiring dialysis or transplantation. For C3G, FABHALTA is used in combination with a renin-angiotensin system (RAS) inhibitor, or as monotherapy if RAS inhibitors are unsuitable or ineffective.
How Does FABHALTA Work?
The active substance iptacopan targets a protein called complement factor B, which plays a central role in the alternative pathway of the complement system. The complement system consists of a cascade of proteins that, when activated, help the body fight infections. However, when this system is dysregulated, it can cause significant tissue damage.
In PNH, red blood cells lack protective surface proteins (CD55 and CD59) that normally shield them from complement attack. As a result, the complement system destroys these unprotected cells, causing chronic hemolytic anemia. By binding to and blocking factor B, iptacopan inhibits the activation of the alternative complement pathway at its amplification loop, thereby preventing complement-mediated destruction of red blood cells. Clinical studies have demonstrated that FABHALTA increases hemoglobin levels and reduces the need for blood transfusions.
In C3G, dysregulation of the alternative complement pathway leads to excessive C3 deposits in the kidneys' glomeruli. By blocking factor B, iptacopan reduces C3 deposition, which in turn decreases proteinuria (protein in the urine) and slows the decline in kidney function. The APPEAR-C3G clinical trial showed that FABHALTA significantly reduced proteinuria compared to placebo after 6 months of treatment.
What Should You Know Before Taking FABHALTA?
Contraindications
You should not take FABHALTA in the following situations:
- Allergy to iptacopan or any other ingredient in the medicine (see the Ingredients section for a full list of excipients).
- Unvaccinated patients: If you have not been vaccinated against Neisseria meningitidis and Streptococcus pneumoniae, you must not start FABHALTA unless your doctor determines that urgent treatment is necessary.
- Active infection with encapsulated bacteria: If you currently have an infection caused by encapsulated bacteria such as Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae type B, treatment with FABHALTA must not be initiated until the infection has been fully resolved.
Warnings and Precautions
FABHALTA increases the risk of life-threatening infections caused by encapsulated bacteria, particularly Neisseria meningitidis (meningococcal disease) and Streptococcus pneumoniae (pneumococcal disease). These infections can progress rapidly and may be fatal. Vaccination does not eliminate the risk entirely. Seek immediate medical attention if you develop fever, headache with stiff neck, rash, confusion, or other signs of infection during treatment.
Before starting FABHALTA, ensure that you receive the following vaccinations:
- Meningococcal vaccine (against Neisseria meningitidis) – covers serogroups A, C, W, Y, and B
- Pneumococcal vaccine (against Streptococcus pneumoniae)
- Haemophilus influenzae type B vaccine – may also be recommended where available
These vaccinations should ideally be given at least 2 weeks before starting treatment. If this timing is not possible and urgent treatment is needed, your doctor will prescribe prophylactic antibiotics to cover the period from treatment initiation until 2 weeks after vaccination. Even after vaccination, it is important to understand that no vaccine provides 100% protection. Your healthcare team will monitor you closely for signs and symptoms of infection throughout your treatment.
Seek immediate medical attention if you experience any of the following symptoms during FABHALTA treatment, as they may indicate a serious infection:
- Fever with or without chills
- Headache with fever, or headache with stiff neck or back
- Fever with skin rash, or fever with chest pain and cough
- Fever with shortness of breath or rapid breathing
- Fever with rapid heart rate
- Nausea or vomiting with headache
- Confusion or altered mental state
- Body aches and flu-like symptoms
- Clammy skin or sensitivity to light
Pregnancy and Breastfeeding
If you are pregnant, breastfeeding, think you might be pregnant, or are planning to have a baby, consult your doctor before taking FABHALTA. It is also important to inform your doctor if you become pregnant during treatment.
The use of FABHALTA during pregnancy requires a careful assessment of the potential benefits and risks by your physician. There is limited data on the effects of iptacopan in pregnant women, and your doctor will discuss the potential risks to you and your unborn child before making a decision about treatment.
It is currently unknown whether iptacopan passes into breast milk or whether it could affect a breastfeeding infant. Your doctor will help you weigh the benefits of breastfeeding for your child against the benefits of continuing treatment with FABHALTA, and will advise you on whether to discontinue breastfeeding or pause treatment.
Children and Adolescents
FABHALTA is not approved for use in children and adolescents under 18 years of age. There is currently no data available on the safety and efficacy of FABHALTA in this age group. Clinical trials in pediatric populations have not been conducted.
Driving and Operating Machinery
FABHALTA has no or negligible effect on the ability to drive vehicles or operate machinery. Patients can generally continue these activities during treatment without restrictions.
How Does FABHALTA Interact with Other Drugs?
Drug interactions are an important consideration with FABHALTA because iptacopan can both be affected by other medications and affect the way other medications work in your body. It is essential to tell your doctor or pharmacist about all medicines you are currently using, have recently used, or might use, including non-prescription and herbal products.
Drugs That Affect FABHALTA
Certain medications can reduce the effectiveness of FABHALTA by altering how it is metabolized in the body. This is clinically important because subtherapeutic levels of iptacopan could lead to breakthrough hemolysis in PNH patients or worsening kidney function in C3G patients.
| Drug | Drug Class | Interaction | Clinical Significance |
|---|---|---|---|
| Rifampicin | Antibiotic (anti-tuberculosis) | May decrease iptacopan plasma levels | High – may reduce FABHALTA efficacy |
Drugs Affected by FABHALTA
FABHALTA may alter the blood levels of several other medications, potentially affecting their efficacy or increasing the risk of side effects. Your doctor may need to adjust doses of these medications or monitor you more closely.
| Drug | Drug Class | Used For | Clinical Action |
|---|---|---|---|
| Carbamazepine | Anticonvulsant | Epilepsy | Monitor levels; dose adjustment may be needed |
| Ciclosporin | Immunosuppressant | Organ transplant rejection | Monitor levels; dose adjustment may be needed |
| Sirolimus | Immunosuppressant | Organ transplant rejection | Monitor levels; dose adjustment may be needed |
| Tacrolimus | Immunosuppressant | Organ transplant rejection | Monitor levels; dose adjustment may be needed |
| Ergotamine | Ergot alkaloid | Migraine | Monitor for ergotamine toxicity |
| Fentanyl | Opioid analgesic | Chronic pain | Monitor for increased sedation or respiratory effects |
| Pimozide | Antipsychotic | Involuntary movements/tics | Monitor for QT prolongation |
| Quinidine | Antiarrhythmic | Abnormal heart rhythm | Monitor cardiac rhythm and drug levels |
| Repaglinide | Meglitinide | Type 2 diabetes | Monitor blood glucose; dose adjustment may be needed |
| Dasabuvir | Antiviral | Hepatitis C | Monitor for increased side effects |
| Paclitaxel | Chemotherapy | Cancer | Monitor for altered chemotherapy efficacy or toxicity |
What Is the Correct Dosage of FABHALTA?
Always take FABHALTA exactly as your doctor or pharmacist has instructed. If you are unsure about any aspect of your dosing, consult your healthcare provider before making any changes. Consistent adherence to the prescribed dosing schedule is essential for maintaining therapeutic drug levels and optimal disease control.
Adults
Standard Adult Dose (PNH and C3G)
200 mg (one capsule) twice daily
- Take one capsule in the morning and one capsule in the evening
- Swallow the capsule whole with a glass of water
- Can be taken with or without food
- Take at the same times each day to help maintain consistent blood levels
For patients with C3G, FABHALTA is typically prescribed in combination with a renin-angiotensin system (RAS) inhibitor such as an ACE inhibitor or angiotensin receptor blocker (ARB). If RAS inhibitors cannot be used or are not effective, FABHALTA may be prescribed as monotherapy. Your doctor will determine the most appropriate treatment regimen for your individual situation.
Children and Adolescents
Pediatric Dose
Not recommended. FABHALTA is not approved for use in patients under 18 years of age. Safety and efficacy have not been established in pediatric populations.
Elderly Patients
Elderly Patients
No dose adjustment is generally required based on age alone. However, elderly patients may have reduced kidney or liver function, and your doctor will consider these factors when prescribing FABHALTA. Regular monitoring of blood counts and renal function is recommended.
Switching from Other PNH Treatments
If you are switching from another PNH treatment (such as eculizumab or ravulizumab) to FABHALTA, your doctor will provide specific instructions on when to start FABHALTA. The timing of the switch is important to ensure continuous complement inhibition and to minimize the risk of breakthrough hemolysis during the transition period. Do not stop your current PNH treatment or start FABHALTA without explicit guidance from your physician.
Duration of Treatment
PNH is a chronic, lifelong condition, and treatment with FABHALTA is typically long-term. Your doctor will schedule regular follow-up appointments to assess treatment effectiveness through blood tests measuring hemoglobin levels, lactate dehydrogenase (LDH), reticulocyte counts, and other hematological parameters. For C3G, treatment duration depends on clinical response, including changes in proteinuria and estimated glomerular filtration rate (eGFR).
Missed Dose
If you miss one or more doses, take a dose of FABHALTA as soon as you remember, even if it is close to the time of your next scheduled dose. Then take your next dose at the usual time. If you have PNH and miss several consecutive doses, contact your doctor promptly, as they may want to monitor you for signs of red blood cell breakdown (hemolysis), such as fatigue, dark urine, or abdominal pain.
Overdose
If you accidentally take more capsules than prescribed, or if someone else accidentally takes your medication, contact your doctor or seek medical attention immediately. There is limited experience with overdose of FABHALTA. Your healthcare provider will assess your condition and provide appropriate supportive care as needed.
If you have PNH, stopping FABHALTA without medical supervision can cause a sudden worsening of your condition. Without complement inhibition, the complement system may rapidly attack your red blood cells, leading to severe hemolytic anemia. If your doctor decides to stop treatment, you will be closely monitored for at least 2 weeks. Symptoms to watch for include significantly low hemoglobin levels, fatigue, blood in urine, abdominal pain, shortness of breath, difficulty swallowing, erectile dysfunction, and blood clots. Contact your doctor immediately if you experience any of these symptoms after stopping treatment.
What Are the Side Effects of FABHALTA?
Like all medicines, FABHALTA can cause side effects, although not everyone experiences them. The side effect profile differs somewhat between PNH and C3G patients, reflecting the different underlying diseases and patient populations. The most important safety concern is the risk of serious infections caused by encapsulated bacteria, which is a class effect of all complement inhibitors.
Side Effects in PNH Patients
Very Common
May affect more than 1 in 10 people
- Upper respiratory tract infections (nose and throat infections)
- Headache
- Diarrhea
Common
May affect up to 1 in 10 people
- Bronchitis (persistent cough or airway irritation)
- Thrombocytopenia (low platelet count, may cause easy bruising or bleeding)
- Dizziness
- Abdominal pain (stomach pain)
- Nausea
- Joint pain (arthralgia)
- Urinary tract infection
Uncommon
May affect up to 1 in 100 people
- Lung infection (pneumonia, may cause chest pain, cough and fever)
- Urticaria (itchy rash, hives)
Side Effects in C3G Patients
Very Common
May affect more than 1 in 10 people
- Upper respiratory tract infections (nose and throat infections)
Common
May affect up to 1 in 10 people
- Pneumococcal infection with pneumonia (lung infection) and sepsis (blood infection)
The side effect data for C3G is based on a smaller clinical trial population compared to PNH. As more patients receive FABHALTA for C3G, additional side effects may be identified. It is important to report any unexpected symptoms to your healthcare provider, even if they are not listed above.
When to Seek Immediate Medical Attention
Contact your doctor or seek emergency medical care immediately if you experience:
- Signs of serious infection (fever, headache with stiff neck, rash, confusion, rapid breathing)
- Signs of significant bleeding or easy bruising (related to low platelet count)
- Signs of severe allergic reaction (difficulty breathing, swelling of face or throat, severe rash)
- Signs of hemolytic crisis if you have PNH (dark urine, severe fatigue, jaundice)
How Should You Store FABHALTA?
Proper storage of medications is important to ensure they remain effective and safe throughout their shelf life. FABHALTA does not require any special storage conditions, making it convenient to store at home. Simply keep the capsules in their original blister packaging at room temperature.
- Keep out of sight and reach of children at all times
- No special temperature requirements – store at room temperature
- Check the expiry date: Do not use FABHALTA after the date marked “EXP” on the carton and blister. The expiry date refers to the last day of that month
- Keep in original packaging to protect from moisture and light
- Proper disposal: Do not throw unused medicine in the drain or with household waste. Return unused capsules to your pharmacy for proper disposal to help protect the environment
What Does FABHALTA Contain?
Active Ingredient
The active substance is iptacopan. Each hard capsule contains 200 mg of iptacopan.
Inactive Ingredients (Excipients)
The other ingredients are:
- Capsule shell: Gelatin, red iron oxide (E172), titanium dioxide (E171), yellow iron oxide (E172)
- Printing ink: Black iron oxide (E172), concentrated ammonia solution (E527), potassium hydroxide (E525), propylene glycol (E1520), shellac (E904)
Appearance and Packaging
FABHALTA capsules are light yellow, opaque hard capsules marked with “LNP200” on the capsule body and “NVR” on the cap. Inside, they contain a white or almost white to light pinkish-violet powder. The capsule is approximately 21–22 mm in size.
FABHALTA is supplied in PVC/PE/PVDC blisters with aluminum foil backing. Available pack sizes include:
- 28 hard capsules
- 56 hard capsules
- Multipack containing 3 cartons of 56 capsules each (168 capsules total)
Not all pack sizes may be marketed in every country. Consult your pharmacist about the pack sizes available in your region.
Marketing Authorization Holder
FABHALTA is marketed by Novartis Europharm Limited, based in Dublin, Ireland. Manufacturing is carried out at facilities in Ljubljana (Slovenia), Barcelona (Spain), and Nuremberg (Germany). For further information about this medicine, contact your local Novartis representative or visit the European Medicines Agency website.
Frequently Asked Questions About FABHALTA
FABHALTA (iptacopan) is used to treat two rare conditions: paroxysmal nocturnal hemoglobinuria (PNH), a blood disorder where the complement system destroys red blood cells causing anemia; and complement factor 3 glomerulopathy (C3G), a kidney disease caused by complement overactivation leading to progressive kidney damage. It is the first oral complement factor B inhibitor, offering a convenient alternative to intravenous complement therapies.
Take one 200 mg capsule twice daily, once in the morning and once in the evening. Swallow the capsule whole with a glass of water. It can be taken with or without food. Taking it at the same times each day helps maintain consistent drug levels. Do not crush, chew, or open the capsule.
Before starting FABHALTA, you must be vaccinated against Neisseria meningitidis (meningococcal disease) and Streptococcus pneumoniae (pneumococcal disease). Vaccination against Haemophilus influenzae type B may also be recommended. These vaccines should be given at least 2 weeks before your first dose. If urgent treatment is needed sooner, your doctor will prescribe prophylactic antibiotics until 2 weeks after vaccination.
In PNH patients, the most common side effects are upper respiratory tract infections, headache, and diarrhea (each affecting more than 1 in 10 patients). Other common effects include bronchitis, low platelet count, dizziness, abdominal pain, nausea, joint pain, and urinary tract infections. In C3G patients, upper respiratory infections are very common, and pneumococcal infections including pneumonia and sepsis are common. The most serious risk is life-threatening infection with encapsulated bacteria.
No, you should never stop FABHALTA without consulting your doctor first. In PNH patients, abruptly stopping complement inhibition can trigger a hemolytic rebound, where the complement system rapidly destroys red blood cells. This can cause severe anemia, dark urine, abdominal pain, shortness of breath, and blood clots. If discontinuation is necessary, your doctor will monitor you closely for at least 2 weeks and may prescribe an alternative complement inhibitor.
Yes, FABHALTA is the first oral monotherapy complement factor B inhibitor approved for PNH. Previous complement inhibitors like eculizumab (Soliris) and ravulizumab (Ultomiris) are monoclonal antibodies that require intravenous infusions every 2-8 weeks in a healthcare setting. FABHALTA's twice-daily oral capsule formulation represents a significant advance in treatment convenience, allowing patients to manage their condition at home.
References
All information on this page is based on peer-reviewed medical literature, regulatory documents, and international clinical guidelines. The following sources were used:
- European Medicines Agency (EMA). FABHALTA (iptacopan) – Summary of Product Characteristics (SmPC). European Public Assessment Report (EPAR). Last updated December 2025.
- Risitano AM, et al. Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: results from the phase 3 APPLY-PNH study. Blood. 2024;143(25):2591-2600.
- Hillmen P, et al. Complement inhibition in paroxysmal nocturnal hemoglobinuria: current landscape and future directions. Blood Reviews. 2023;61:101084.
- Bomback AS, et al. Iptacopan for C3 glomerulopathy: results from the phase 3 APPEAR-C3G trial. New England Journal of Medicine. 2024;391(10):905-916.
- Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124(18):2804-2811.
- World Health Organization (WHO). Model List of Essential Medicines. 23rd edition. Geneva: WHO; 2023.
- Novartis Pharmaceuticals. FABHALTA (iptacopan) Prescribing Information. 2024.
- Smith RJH, et al. C3 glomerulopathy: understanding a rare complement-driven glomerulonephritis. Nature Reviews Nephrology. 2019;15(3):129-143.
Medical Editorial Team
This article has been written and reviewed by our team of medical specialists with expertise in hematology, nephrology, and clinical pharmacology. All content follows our strict editorial standards and is based on the latest clinical evidence.
iMedic Medical Editorial Team – specialists in clinical pharmacology and rare blood diseases. Content created following GRADE evidence framework and international medical guidelines.
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