Pegcetacoplan

Pegcetacoplan is a prescription medicine used for the treatment of paroxysmal nocturnal hemoglobinuria.

• Pegcetacoplan is available under the following different brand names: Empaveli

Common side effects of Pegcetacoplan include:

• injection-site reactions (redness, swelling, hard lump, bruising, itching, warmth, pain, rash),
• infections,
• diarrhea,
• abdominal pain,
• respiratory tract infection,
• viral infection,
• chest pain,
• back pain,
• headache, and
• high blood pressure (hypertension).

Serious side effects of Pegcetacoplan include:

• Serious infections
• Infusion-related reactions

Rare side effects of Pegcetacoplan include:

• none

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out. 

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Injection, solution

• 1,080 mg/20mL (54mg/mL) single-dose vial

Paroxysmal Nocturnal Hemoglobinuria

Adult dosage

• 1,080 mg SC infusion twice weekly
• Switching to pegcetacoplan from C5 inhibitors
• Switching from eculizumab: Initiate pegcetacoplan while continuing eculizumab at its current dose; after 4 weeks, discontinue eculizumab before continuing on monotherapy with pegcetacoplan
• Switching from ravulizumab: Initiate pegcetacoplan no more than 4 weeks after the last ravulizumab dose

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Pegcetacoplan has no noted severe interactions with any other drugs.
• Pegcetacoplan has no noted serious interactions with any other drugs.
• Pegcetacoplan has no noted moderate interactions with any other drugs.
• Pegcetacoplan has no noted minor interactions with any other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, or if you have health questions or concerns.

Contraindications

• Hypersensitivity to pegcetacoplan or any of the excipients
• Patients not vaccinated against certain encapsulated bacteria, unless the risks of delaying pegcetacoplan treatment outweigh the risks of developing a bacterial infection with an encapsulated organism
• Patients with unresolved serious infection caused by encapsulated bacteria, including S pneumoniae, N meningitidis, and H influenzae

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Pegcetacoplan?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Pegcetacoplan?”

Cautions

• Available only through a restricted access program
• Infusion-related reactions reported, including systemic hypersensitivity reactions (eg, facial swelling, rash, urticaria); discontinue infusion immediately and provide appropriate treatment, per standard of care, if severe hypersensitivity reaction, including anaphylaxis, occurs; monitor until signs and symptoms resolve
• Serious infections caused by encapsulated bacteria
• May increase the risk of serious infections caused by encapsulated bacteria
• All patients must be vaccinated against these bacteria according to the most current ACIP recommendations for patients with altered immunocompetence associated with complement deficiencies
• Revaccinate patients following ACIP recommendations considering the duration of therapy
• If vaccination history is unknown, administer required vaccines at least 2 weeks before the first pegcetacoplan dose
• If immediate therapy with pegcetacoplan is indicated, administer the required vaccine as soon as possible and provide patients with 2 weeks of antibacterial drug prophylaxis
• Closely monitor for infection
• Monitoring after discontinuation
  • Closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with a sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms (eg, fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events [including thrombosis], dysphagia, erectile dysfunction)
  • Monitor for at least 8 weeks to detect hemolysis and other reactions
  • If hemolysis, including elevated LDH, occurs, consider restarting treatment
• Interference with laboratory tests
• Drug may interfere with silica reagents in coagulation panels that result in artificially prolonged activated partial thromboplastin time; avoid silica reagents in coagulation panels

Pregnancy and Lactation

• Insufficient data regarding use in pregnant females to inform of drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes
• There are risks to the mother and fetus associated with untreated PNH during pregnancy
• Clinical considerations
  • PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages, and increased maternal mortality, and adverse fetal outcomes, including fetal death and premature delivery
• Contraception
  • May cause embryofetal harm when administered to pregnant females
  • Pregnancy testing recommended for females of reproductive potential before treatment
  • Advise female patients of reproductive potential to use effective contraception during treatment and for 40 days after the last dose
• Lactation
  • Unknown if secreted in human milk or whether there is potential for absorption and harm to the infant
  • There are no data on the effects on milk production
  • Pegcetacoplan is present in the milk of lactating monkeys; since many medicinal products are secreted into human milk, and the potential exists for serious adverse reaction in a breastfeeding child, breastfeeding should be discontinued during treatment and for 40 days after the last dose