Pegcetacoplan

What is Pegcetacoplan?

• Pegcetacoplan (EMPAVELI) is a complement inhibitor indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

What are the uses of this medicine?

• This medicine is used for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

How does this medicine work?

• A pegylated peptide inhibitor of human complement components c3 and c3b, with potential use as a treatment for various diseases in which excessive complement activation plays a key role, including paroxysmal nocturnal hemoglobinuria (pnh), geographic atrophy (ga), cold agglutinin disease (cad), and c3 glomerulopathy (c3g).
• Upon administration, pegcetacoplan selectively binds to c3 and c3b and inhibits their activities.
• This prevents complement pathway activation, and inhibits complement-mediated inflammation and cell lysis.
• Excessive complement activation plays a key role in various inflammatory and autoimmune diseases, and leads to tissue destruction.
• C3 is a key component of the complement system, and the complement system is an integral component of the innate immune response.

Who Should Not Use this medicine ?

This medicine cannot be used in pateints:

• with hypersensitivity to pegcetacoplan or to any of the excipients.
• who are not currently vaccinated against certain encapsulated bacteria, unless the risks of delaying EMPAVELI treatment outweigh the risks of developing a bacterial infection with an encapsulated organism.
• with unresolved serious infection caused by encapsulated bacteria including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae.

Is this medicine FDA approved?

• It was approved for use in the United States in 2021.

How should this medicine be used?

• Vaccinate patients against encapsulated bacteria, including Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B at least 2 weeks prior to initiation of EMPAVELI therapy.
• Provide 2 weeks of antibacterial drug prophylaxis to patients if EMPAVELI must be initiated immediately and vaccines are administered less than 2 weeks before starting therapy with EMPAVELI.

Recommended dosage

• Recommended dosage is 1,080 mg by subcutaneous infusion twice weekly via a commercially available pump.

Dosage for patients switching to EMPAVELI from C5 inhibitors To reduce the risk of hemolysis with abrupt treatment discontinuation:

• For patients switching from eculizumab, initiate EMPAVELI while continuing eculizumab at its current dose. After 4 weeks, discontinue eculizumab before continuing on monotherapy with EMPAVELI.
• For patients switching from ravulizumab, initiate EMPAVELI no more than 4 weeks after the last dose of ravulizumab.

Administration

• EMPAVELI is for subcutaneous infusion using an infusion pump.
• EMPAVELI is intended for use under the guidance of a healthcare professional. After proper training in subcutaneous infusion, a patient may self‑administer, or the patient’s caregiver may administer EMPAVELI, if a healthcare provider determines that it is appropriate.
• Refer to the EMPAVELI Instructions for Use and the infusion pump manufacturer’s instructions for full preparation and administration information.
• Use aseptic technique when preparing and administering EMPAVELI.
• Prior to use‚ allow EMPAVELI to reach room temperature for approximately 30 minutes. Keep the vial in the carton until ready for use to protect from light.
• Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. EMPAVELI is a clear, colorless to slightly yellowish solution. Do not use if the liquid looks cloudy, contains particles, or is dark yellow.
• Use a needleless transfer device (such as a vial adapter) or a transfer needle to fill the syringe.
• Rotate infusion sites (i.e., abdomen, thighs, hips, upper arms) from one infusion to the next. Do not infuse where the skin is tender, bruised, red, or hard. Avoid infusing into tattoos, scars, or stretch marks.
• If multi-infusion sets are needed, ensure the infusion sites are at least 3 inches apart.
• The typical infusion time is approximately 30 minutes (if using two infusion sites) or approximately 60 minutes (if using one infusion site).
• Discard any unused portion.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Injection: 1,080 mg/20 mL (54 mg/mL) in a single‑dose vial.

This medicine is available in fallowing brand namesː

• EMPAVELI

What side effects can this medication cause?

The most common side effects of this medicine include:

• injection‑site reactions
• infections
• diarrhea
• abdominal pain
• respiratory tract infection
• viral infection
• fatigue

What special precautions should I follow?

• The use of EMPAVELI may predispose individuals to serious, life‑threatening, or fatal infections caused by encapsulated bacteria including Streptococcus pneumoniae , Neisseria meningitidis types A, C, W, Y, and B, and Haemophilus influenzae type B (Hib).
• To reduce the risk of infection, all patients must be vaccinated against these bacteria according to the most current ACIP recommendations.
• For patients without known history of vaccination, administer required vaccines at least 2 weeks prior to receiving the first dose of EMPAVELI.
• Monitor patients for infusion‑related reactions and institute appropriate medical management as needed.
• Because of the risk of serious infections, EMPAVELI is available only through a restricted program under a REMS. Under the EMPAVELI REMS, prescribers must enroll in the program.
• There may be interference between silica reagents in coagulation panels and EMPAVELI that results in artificially prolonged activated partial thromboplastin time (aPTT); therefore, avoid the use of silica reagents in coagulation panels.
• After discontinuing treatment with EMPAVELI, closely monitor for signs and symptoms of hemolysis, identified by elevated LDH levels along with sudden decrease in PNH clone size or hemoglobin, or reappearance of symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, major adverse vascular events (including thrombosis), dysphagia, or erectile dysfunction.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are insufficient data on EMPAVELI use in pregnant women to inform a drug‑associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.

Can this medicine be used in children?

• Safety and effectiveness have not been established.

What should I know about storage and disposal of this medication?

Store vials of EMPAVELI refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not use beyond the expiration date stamped on the carton.

• Dailymed label info on Pegcetacoplan
• FDA Pegcetacoplan

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Latest articles - Pegcetacoplan

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Source: Data courtesy of the U.S. National Library of Medicine. Since the data might have changed, please query MeSH on Pegcetacoplan for any updates.

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