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Empaveli
Published
Product name
Empaveli
Active ingredient
Pegcetacoplan
Submission type
New chemical entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Empaveli (pegcetacoplan) was approved for the following therapeutic use:
Empaveli is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have an inadequate response to, or are intolerant of, a C5 inhibitor.
How this medicine works
Pegcetacoplan binds to complement protein C3 (also known as complement component 3) and its activation fragment C3b with high affinity, thereby regulating the cleavage of C3 and the generation of downstream effectors of complement activation. In paroxysmal nocturnal haemoglobinuria (PNH), extravascular haemolysis (EVH) is facilitated by C3b opsonization while intravascular haemolysis (IVH) is mediated by the downstream membrane attack complex (MAC). Pegcetacoplan exerts broad regulation of the complement cascade by acting proximal to both C3b and MAC formation, thereby controlling the mechanisms that lead to EVH and IVH. These functions of pegcetacoplan underlie the observed sustained reduction in complement-mediated haemolytic activity in patients with PNH.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Empaveli was considered favourable for the therapeutic use approved.
Sponsor