ES 
EN 
FABHALTA® (Iptacopan)
Iptacopan (Fabhalta) was approved by the Food and Drug Administration (FDA) in March 2025 to reduce proteinuria in adults with complement 3 glomerulopathy (C3G). It was the first medication specifically approved for the treatment of C3G.
Medically reviewed by Dr. John Sperati, MD
How Does FABHALTA® Work for C3G?
In C3G, part of the immune system, called the alternative pathway of complement, is too active and injures the kidneys. Iptacopan (Fabhalta) works by reducing the activity of this pathway. To reduce the activity, iptacopan binds to and prevents the function of a protein called complement factor B (CFB). CFB is important for the activation of the alternative pathway of complement. By preventing CFB from functioning properly, the alternative pathway of complement is less active. Making this pathway less active reduces the ability of the complement system to cause kidney injury in people with C3G.
Cómo es FABHALTA® for C3G Administered?
Iptacopan (Fabhalta) is a pill taken twice a day by mouth. It should be swallowed whole and can be taken either with or without food.
Is FABHALTA® for C3G Right for Me?
You should discuss with your doctor if iptacopan (Fabhalta) is right for you. Iptacopan (Fabhalta) is approved for the treatment of C3G, a disease that requires a kidney biopsy for diagnosis. Iptacopan (Fabhalta) has not been specifically approved for people younger than 18 years of age. Like all medications, iptacopan (Fabhalta) has potential side-effects that should be discussed and considered in the context of your medical conditions.
*Nota: La decisión de recetar un medicamento es responsabilidad de su médico/equipo médico en función de la evaluación de su afección. Lo anterior está destinado únicamente a fines informativos. Discuta esta información y toda la información sobre drogas/medicamentos con su médico antes de comenzar o suspender cualquier medicamento.
Preguntas frecuentes
Iptacopan (Fabhalta) was approved based on results from the APPEAR clinical trial, a randomized, double blind Phase 3 trial of iptacopan versus placebo (dummy pill). Participants enrolled in the trial had to be 18 years or older, have an eGFR ≥30 ml/min/1.73m2, urine protein to creatinine ratio ≥1 gram/gram, and a C3 level <77 mg/dL. Participants with kidney transplants were not included in this study. After 6 months, participants receiving iptacopan had an approximately 35% greater reduction in urine protein than those receiving placebo. Data from the APPEAR trial and other studies demonstrate iptacopan slows the loss of kidney function over 12 months.
The only other FDA approved treatment for C3G is pegcetacoplan (Empaveli). Here is how they differ.
How they are taken:
- Pegcetacoplan (Empaveli) is an under-the-skin infusion administered at home twice a week.
- Iptacopan (Fabhalta) is a pill taken by mouth twice a day.
FDA approval:
- Pegcetacoplan (Empaveli) is approved for people aged 12 years and older with C3G or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria and the loss of kidney function.
- Iptacopan (Fabhalta) is approved for adults aged 18 years and older with C3G to reduce proteinuria.
How they work:
The complement system has different pathways that can be overactive in people with C3G. These pathways are called: alternative, classical and lectin.
- Iptacopan mostly reduces the activity of the alternative pathway. It binds and reduces the function of protein complement factor B (CFB).
- Pegcetacoplan works to reduce the activity of three pathways of the complement system. It binds and reduces the function of a protein called complement C3. Complement C3 is important to all three complement pathways (alternative, classical and lectin).
Iptacopan (Fabhalta) has been shown to have an acceptable safety profile in clinical trials and real-world use. By reducing the activity of the complement system (part of the immune system), iptacopan (Fabhalta) may increase your risk for infection. In particular, the risk for infection with encapsulated bacteria such as Neisseria species, Streptococcus pneumoniae, y Haemophilus influenzae Type B is increased. These infections can be serious and lead to death if untreated. To help reduce the risk of infection, you must receive a series of vaccines. Some people may also need to take prophylactic (or preventative) antibiotics during iptacopan (Fabhalta) treatment.
There are two types of medications used to treat C3G: those that suppress the immune system (immunosuppressants) and those that do not suppress the immune system (non-immunosuppressive medications).
Non-immunosuppressive medications (also called “foundational” medications) are often used to:
- treat high blood pressure
- lower urine protein
- slow the accumulation of scar tissue in the kidneys that leads to loss of function
These therapies include blood pressure medications like angiotensin converting enzyme inhibitors (ACE inhibitors) or angiotensin receptor blockers (ARB), sodium-glucose transporter 2 inhibitors (SGLT2 inhibitors), and mineralocorticoid receptor antagonists (MRA).
Non-immunosuppressive medications do not increase the risk for infection, but they also do not directly target the overactive complement system that injures kidneys in C3G.
Immunosuppressant medications:
Both FDA approved medications for the treatment of C3G, iptacopan (Fabhalta) and pegcetacoplan (Empaveli), are immunosuppressant medications. Both medications target the specific part of the immune system that leads to kidney damage in C3G.
Other immune suppressing therapies are sometimes used to treat C3G. This can include oral immune suppressants such as mycophenolate mofetil and prednisone (a steroid). These therapies reduce the immune system in general and may help to limit kidney injury. They do not specifically target the part of the immune system that is overactive in C3G. Some people with C3G form antibodies (other proteins in the immune system) that cause the complement system to be too active and injure kidneys. Immunosuppressants like mycophenolate mofetil and prednisone may reduce antibodies so they do not trigger the complement system. Like any immune suppressant, these therapies can increase the risk for infection.
Iptacopan (Fabhalta) is available only by prescription from your physician. If you take Fabhalta, both you and your prescribing doctor are required to enroll in a Risk Evaluation and Mitigation Strategy (REMS) program. You will need to receive a series of vaccines at least 2 weeks prior to starting the medication. The REMS program helps to ensure you are aware of the infection risks associated with iptacopan (Fabhalta) and have been educated on how to identify and respond to signs or symptoms of infection. Pharmaceutical assistance programs might be available to help you obtain or afford the medication.
Encuentre un especialista
Es importante trabajar con un nefrólogo que se especialice en enfermedades renales raras (ERR) y que pueda brindarle asesoramiento personalizado según sus necesidades de salud individuales y los cambios en los tratamientos disponibles. NephCure tiene una lista seleccionada de especialistas de NephCure en todo el país listos para ayudarlo.
Ensayos clínicos
Desde acceder a tratamientos de vanguardia hasta ser parte de investigaciones innovadoras, descubra los beneficios que pueden marcar una diferencia real. Obtenga más información sobre los ensayos clínicos que se están llevando a cabo actualmente y encuentre oportunidades cerca de usted.
