Pegcetacoplan

FDA approved

Pegcetacoplan is a complement C3 inhibitor. As Empaveli (FDA approved 2021) it treats paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder; as Syfovre (2023) it is injected into the eye for geographic atrophy.

At a glance

Where it acts: Heart & blood
Regulatory status: FDA approvedApproved by the U.S. FDA for at least one medical use.

Mechanism of Action

Pegcetacoplan is a pair of cyclic peptides joined by a PEG chain. It binds complement protein C3 and blocks the complement cascade upstream, preventing immune destruction of red blood cells in PNH.

Typical Dosage (Research)

Prescription-only medicine. Dosing is set by a physician according to the FDA-approved label and the condition being treated.

Side Effects & Risks

Injection site reactions, infections, diarrhea, abdominal pain and fatigue.

Serious infections from encapsulated bacteria (meningococcus, pneumococcus, Haemophilus); vaccination is required and it carries a boxed warning. Eye injections carry risks of inflammation and wet AMD.

Who Uses Pegcetacoplan

Adults with PNH; adults with geographic atrophy secondary to age-related macular degeneration.