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Pegcetacoplan: First Approval.

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Pegcetacoplan is a novel C3-targeted therapy approved for paroxysmal nocturnal haemoglobinuria (PNH). This drug effectively targets complement component 3 (C3), offering a new treatment option for PNH patients.

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Area of Science:

  • Pharmacology
  • Immunology
  • Hematology

Background:

  • Pegcetacoplan is a PEGylated pentadecapeptide targeting complement component 3 (C3).
  • It addresses complement-mediated diseases by controlling C3 cleavage and downstream effector generation.
  • This includes both extravascular and intravascular hemolysis pathways.

Purpose of the Study:

  • To summarize the development of pegcetacoplan for treating complement-mediated diseases.
  • To highlight the first approval of pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH).

Main Methods:

  • Pegcetacoplan binds to complement component 3 (C3) and C3b.
  • It inhibits C3 cleavage, thereby controlling complement activation.
  • Development milestones leading to regulatory approval were reviewed.

Main Results:

  • Pegcetacoplan received US FDA approval in May 2021 as the first C3-targeted therapy for PNH.
  • It is indicated for adults with PNH, including those switching from C5 inhibitors.
  • Regulatory assessments are ongoing in the EU and Australia.

Conclusions:

  • Pegcetacoplan represents a significant advancement in PNH treatment by targeting C3.
  • The drug's potential in other complement-mediated diseases like AMD and C3 glomerulopathy is under investigation.
  • The approved dosage is 1080 mg twice weekly via subcutaneous infusion.