Pronunciation:

ip-ta-KOE-pan

Brand Names:

• Fabhalta

Dosage Forms:

• Capsule

Warnings:

Warning: Serious Infections Caused By Encapsulated Bacteria Iptacopan a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B. Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of iptacopan, unless the risks of delaying therapy with iptacopan outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor. See Warnings and Precautions (5.1) for additional guidance on the management of the risk of serious infections caused by encapsulated bacteria. Patients receiving iptacopan are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, iptacopan is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the FABHALTA REMS

Classifications:

Therapeutic—

Blood Modifier Agent

Uses of This Medicine:

Iptacopan is used to treat a type of blood disease called paroxysmal nocturnal hemoglobinuria (PNH). This medicine helps reduce red blood cell destruction or breakdown (hemolysis) in patients with PNH.

This medicine is available only under a restricted distribution program called Fabhalta® REMS (Risk Evaluation and Mitigation Strategy) Program.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of iptacopan in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of iptacopan in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Carbamazepine
• Gemfibrozil
• Ivosidenib
• Rifampin

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Bacterial infections (eg, pneumonia, meningitis, or flu-like infection), unresolved and serious—Should not be given to patients with these conditions.

• Infection or
• Weakened immune system—Use with caution. This medicine may decrease your body’s ability to fight infection.

• Kidney disease, severe or
• Liver disease, severe—Use is not recommended in patients with these conditions.

Proper Use of This Medicine:

Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.

It is very important that you understand the requirements of the Fabhalta® REMS program, and become familiar with the Fabhalta® medication guide. Read and follow these instructions carefully. Ask your doctor if you have any questions. Ask your pharmacist for the medication guide if you do not have one.

You may take this medicine with or without food.

Swallow the capsule whole. Do not open, break, or chew it.

Tell your doctor if you are changing treatment from eculizumab or ravulizumab to Fabhalta®.

• Switching from eculizumab to Fabhalta®: You should start taking Fabhalta® no later than 1 week after your last dose of eculizumab.
• Switching from ravulizumab to Fabhalta®: You should start taking Fabhalta® no later than 6 weeks after your last dose of ravulizumab.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage form (capsule):
  • For paroxysmal nocturnal hemoglobinuria (PNH):
    • Adults—200 milligrams (mg) 2 times a day.
    • Children—Use and dose must be determined by your doctor.

Missed dose—

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If a dose is missed, take it as soon as possible even if it is almost time for your next dose. Then, take the next dose at the regular scheduled time.

Storage—

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure this medicine is working properly. Blood and urine tests may be needed to check for unwanted effects.

Iptacopan may increase your chance of having serious infections, including Streptococcus pneumoniae, Neisseria meningitidis, or Haemophilus influenzae. Avoid people who are sick or have infections. Tell your doctor right away if you develop headaches, nausea, vomiting, fever, a stiff neck or back, a rash, confusion, muscle aches, or if your eyes have become sensitive to light. Make sure you have received a vaccine to prevent encapsulated bacterial infections at least 2 weeks before you receive this medicine. You may also be given antibiotic medicines to prevent infections if you are to use this medicine right away. If you have already received the vaccine in the past, your doctor will decide if you need another dose.

Ask your doctor for a patient safety card. This card will list the symptoms of encapsulated bacterial infections and what to do if you have them. Carry the card with you at all times during treatment and for 2 weeks after your last dose. You will need to show the card to any doctor who treats you.

When this medicine is stopped you may develop red blood cell destruction or breakdown (hemolysis). Your doctor will need to monitor you closely for at least 2 weeks after you stop using this medicine. Be sure to keep all appointments.

This medicine may increase your cholesterol and triglycerides (fats). Talk with your doctor if you have any concerns about this.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Black, tarry stools

bladder pain

bleeding gums

bloody or cloudy urine

blurred vision

body aches or pain

burning, itching, and pain in hairy areas, pus at root of the hair

chest pain or tightness

chills

confusion

cough

cough producing mucus

diarrhea

dizziness

ear congestion

fainting

fever

frequent urge to urinate

general feeling of discomfort or illness

headache

indigestion

joint pain

lightheadedness

loss of taste or smell

loss of voice

lower back or side pain

muscle aches

nervousness

painful blisters on trunk of the body

painful or difficult urination

pinpoint red spots on the skin

pounding in the ears

rapid, shallow breathing

severe nausea or vomiting

shivering

slow or fast heartbeat

sneezing

sore throat

stomach pain

stuffy or runny nose

trouble breathing

trouble sleeping

unusual bleeding or bruising

unusual tiredness or weakness

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Difficulty in moving

joint pain

muscle pain or stiffness

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.