Pegcetacoplan (Subcutaneous)

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

This medicine is available only through a restricted distribution program called Empaveli™ REMS (Risk Evaluation and Mitigation Strategy) Program.

It is very important that your doctor checks 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.

Using this medicine could harm your unborn baby. If you are a woman who can get pregnant, your doctor may do tests to ensure you are not pregnant before starting this treatment. Use an effective form of birth control during treatment with this medicine and for 40 days after the last dose to avoid getting pregnant. Tell your doctor right away if you think you have become pregnant.

Pegcetacoplan may increase your chance of having serious infections, including Streptococcus pneumoniae, Neisseria meningitidis, and 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 for 2 weeks 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 months after your last dose. You will need to show the card to any doctor who treats you.

This medicine may cause infusion-related reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you start to have a fever, chills or shaking, dizziness, chest pain, trouble breathing, itching or rash, lightheadedness, fainting, or swelling of the face, tongue, or throat after receiving this medicine.

When this medicine is stopped, you could have red blood cell destruction or breakdown (hemolysis). Your doctor will need to monitor you closely for at least 8 weeks after you stop using this medicine. Be sure to keep all appointments.

Before you have any medical tests, tell the medical doctor in charge that you are taking this medicine. The results of some tests may be affected by this medicine.

In deciding to use a medicine, you and your doctor must consider the risks of taking the medicine against the benefits it will provide. For this medicine, keep the following in mind:

Allergies

Inform your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also, tell your healthcare 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.

Pediatric

No appropriate studies have been done on how age affects the effects of pegcetacoplan injection in children. Safety and effectiveness have not been established.

Geriatric

No appropriate studies have shown geriatric-specific problems that would limit the usefulness of pegcetacoplan injection in older adults.

Breast Feeding

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

Interactions with Medicines

Some medicines should not be used together at all, while in other cases, two different medicines may be used together even if an interaction could happen. In these situations, your doctor may want to adjust the dose or take other precautions. Inform your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Interactions with Food/Tobacco/Alcohol

Some medicines should not be taken at or around the time of eating food or certain types of food due to possible interactions. Using alcohol or tobacco with some medicines may also lead to interactions. Discuss with your healthcare professional about using your medicine with food, alcohol, or tobacco.

Other Medical Problems

Having other medical problems may affect your use of this medicine. Make sure to inform your doctor if you have any other medical issues, especially:

• Bacterial infections (e.g., pneumonia, meningitis, and flu-like infections), encapsulated—Should not be given to patients with these conditions.

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

• No current vaccination against encapsulated bacterial infections—Should not be given to patients with this condition unless the doctor decides that urgent treatment is needed.

Along with its needed effects, a medicine may cause some unwanted effects. Not all of these side effects may happen, but if they do, they may need medical attention.

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

More common

• Black, tarry stools
• bleeding, blistering, burning, coldness, discoloration of the skin, feeling of pressure, hives, infection, inflammation, itching, lumps, numbness, pain, rash, redness, scarring, soreness, stinging, swelling, tenderness, tingling, ulceration, or warmth at the injection site
• bleeding gums
• bloating or swelling of the face, arms, hands, lower legs, or feet
• blood in the urine or stools
• bloody vomit
• blurred vision
• body aches or pain
• change in the color, amount, or odor of the vaginal discharge
• chest pain, discomfort, or tightness
• chills
• confusion
• cough or hoarseness
• cough producing mucus
• decreased urine
• diarrhea
• difficulty in breathing
• dizziness
• dry mouth
• ear congestion
• fainting
• fever
• flu-like symptoms
• general feeling of discomfort or illness
• headache
• increased thirst
• irregular heartbeat
• lightheadedness
• loss of appetite
• loss of voice
• mood changes
• muscle aches, pain, or cramps
• nausea
• numbness or tingling in the hands, feet, or lips
• pinpoint red spots on the skin
• pounding in the ears
• rapid, shallow breathing
• rapid weight gain
• seizures
• slow or fast heartbeat
• sneezing
• sore throat
• severe stomach pain
• sleepiness
• stuffy or runny nose
• thickening of bronchial secretions
• tingling of the hands or feet
• trouble breathing
• tooth or gum pain
• unusual bleeding or bruising
• unusual tiredness or weakness
• unusual weight gain or loss

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. Your health care professional may also 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, are bothersome, or if you have any questions about them:

More common

• Arm or leg pain
• bloody nose
• diarrhea
• difficulty in moving
• heartburn
• joint pain
• lack or loss of strength
• large, flat, blue or purplish patches in the skin
• pain in the lower back, bottom, hips, or upper legs
• unusual drowsiness, dullness, or feeling of sluggishness

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.

In the U.S.

• Empaveli

Available Dosage Forms:

• Solution

Therapeutic Class: Blood Modifier Agent

A nurse or other trained health professional may give you this medicine. It is given through a needle placed under your skin, usually in the stomach, thighs, hips or upper arm. It must be given slowly, so the needle will have to stay in place using an infusion pump for at least 30 minutes (for 2 infusion sites) or at least 60 minutes (for 1 infusion site).

You or your caregiver may be trained to prepare and inject this medicine at home. Be sure that you understand how to use the medicine and the infusion pump.

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

If you use this medicine at home, you will be shown the body areas where the infusion can be given. Use a different body area each time you give yourself an injection. Keep track of where you give each injection to make sure you rotate body areas. This will help prevent skin problems. Do not inject into areas where the skin is tender, red, bruised, or hard or in areas with tattoos, scars, or stretch marks.

Allow this medicine to warm to room temperature for 30 minutes before using it. Do not warm it in any other way.

Check the liquid in the vial. It should be clear and colorless to slightly yellow solution. Do not use the medicine if the liquid is cloudy, discolored, or has particles in it.

Tell your doctor if you are changing treatment from eculizumab or ravulizumab to Empaveli™.

• Switching from eculizumab to Empaveli™: At first, you should start receiving Empaveli™ while you continue your treatment with eculizumab. Then after 4 weeks, you should stop receiving eculizumab before you continue your treatment with Empaveli™.
• Switching from ravulizumab to Empaveli™: You should start receiving Empaveli™ 4 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 injectable dosage form (solution):For paroxysmal nocturnal hemoglobinuria (PNH): Adults—1080 milligrams (mg) injected under the skin 2 times a week. Children—Use and dose must be determined by your doctor.

Missed Dose

Call your doctor or pharmacist for instructions.

If you miss a dose of this medicine, use it as soon as possible. Do not double doses.

Storage

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.

Store in the refrigerator. Do not freeze.

Keep the medicine in its original carton, away from direct light, until you are ready to use it. Throw away any unused medicine.

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