Pegcetacoplan
Pegcetacoplan is a complement inhibitor that targets complement protein C3 and its activation fragment C3b to regulate complement-mediated cell damage. It is primarily used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by the destruction of red blood cells due to uncontrolled complement activation.
Pegcetacoplan is an immunosuppressant that inhibits complement C3 to treat paroxysmal nocturnal hemoglobinuria (PNH) by preventing red blood cell destruction.
Key facts at a glance
- ATC
- L04AJ03
- RxNorm
- 2557372
- Generic
- Pegcetacoplan
- Class
- atc-l04
- Brands
- Empaveli, Syfovre
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
6 groupspregnancy
all trimestersavoid if possible
use only if potential benefits outweigh potential risks to the fetus
regular fetal monitoring recommended
Use with caution and monitor for adverse effects.
lactation
breastfeedingdiscontinue or avoid
not recommended during breastfeeding due to unknown excretion in breast milk
monitor infant for signs of infection
geriatric
elderlyno specific adjustment needed
use with caution due to increased susceptibility to infections
monitor renal function and for signs of infection
Use with caution and monitor for adverse effects.
pediatric_age
children adolescentsnot recommended
not established for use in pediatric patients under 18 years of age
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
renal_impairment
mild to moderateno initial adjustment required
use with caution, monitor renal function regularly
monitor renal function tests periodically
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno specific guidance available
use with caution due to limited clinical data
monitor liver function tests periodically
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
Empaveli approved for paroxysmal nocturnal hemoglobinuria
Drug class
Pharmacological classification
FDA-approved indications
Therapeutic indications
Mechanism of action
Pharmacodynamics
Pegcetacoplan is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult patients. It helps reduce hemolysis and the need for blood transfusions in patients with this rare, acquired blood disorder.
Pegcetacoplan is a pegylated peptide that binds with high affinity to complement protein C3 and C3b, inhibiting cleavage of C3 and preventing generation of downstream complement effectors that cause cell lysis and inflammation.
This medication is administered subcutaneously twice weekly. Patients should be monitored for infections, including meningococcal infections, as complement inhibition increases susceptibility. Vaccination against encapsulated bacteria is recommended prior to initiation. Common side effects include injection site reactions, infections, and fatigue.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy