pegcetacoplan

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pegcetacoplan

GtoPdb Ligand ID: 13826

Synonyms: APL-2 | Empaveli®

pegcetacoplan is an approved drug

Comment: Pegcetacoplan is a compstatin family complement factor C3 inhibitor [6]. It contains two identical 12 amino acid compstatin-derived cyclic peptides [Ac-Ile-Cys(1)-Val-(Me)Trp-Gln-Asp-Trp-Gly-Ala-His-Cys(1)-Thr-NH₂] that are bridged by a 40 kDa PEG moiety at their amidated carboxy termini to improve circulating half-life [4-5].

References
1. Barcellini W, Fattizzo B, Zaninoni A. (2018) Current and emerging treatment options for autoimmune hemolytic anemia. Expert Rev Clin Immunol, 14 (10): 857-872. [PMID:30204521]
2. Heier JS, Lad EM, Holz FG, Rosenfeld PJ, Guymer RH, Boyer D, Grossi F, Baumal CR, Korobelnik JF, Slakter JS et al.. (2023) Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials. Lancet, 402 (10411): 1434-1448. [PMID:37865470]
3. Hoy SM. (2021) Pegcetacoplan: First Approval. Drugs, 81 (12): 1423-1430. [PMID:34342834]
4. Lamers C, Mastellos DC, Ricklin D, Lambris JD. (2022) Compstatins: the dawn of clinical C3-targeted complement inhibition. Trends Pharmacol Sci, 43 (8): 629-640. [PMID:35090732]
5. Mastellos DC, Ricklin D, Sfyroera G, Sahu A. (2022) From discovery to approval: A brief history of the compstatin family of complement C3 inhibitors. Clin Immunol, 235: 108785. [PMID:34147650]
6. Ricklin D, Mastellos DC, Lambris JD. (2019) Therapeutic targeting of the complement system. Nat Rev Drug Discov, [Epub ahead of print]. [PMID:31819218]
7. Xu B. (2023) Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria. Clin Exp Med, 23 (3): 717-726. [PMID:35441351]
8. (2024) Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria. N Engl J Med, 390 (11): 1060. [PMID:38478008]

References

1. Barcellini W, Fattizzo B, Zaninoni A. (2018)
Current and emerging treatment options for autoimmune hemolytic anemia.
Expert Rev Clin Immunol, 14 (10): 857-872. [PMID:30204521]

2. Heier JS, Lad EM, Holz FG, Rosenfeld PJ, Guymer RH, Boyer D, Grossi F, Baumal CR, Korobelnik JF, Slakter JS et al.. (2023)
Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials.
Lancet, 402 (10411): 1434-1448. [PMID:37865470]

3. Hoy SM. (2021)
Pegcetacoplan: First Approval.
Drugs, 81 (12): 1423-1430. [PMID:34342834]

4. Lamers C, Mastellos DC, Ricklin D, Lambris JD. (2022)
Compstatins: the dawn of clinical C3-targeted complement inhibition.
Trends Pharmacol Sci, 43 (8): 629-640. [PMID:35090732]

5. Mastellos DC, Ricklin D, Sfyroera G, Sahu A. (2022)
From discovery to approval: A brief history of the compstatin family of complement C3 inhibitors.
Clin Immunol, 235: 108785. [PMID:34147650]

6. Ricklin D, Mastellos DC, Lambris JD. (2019)
Therapeutic targeting of the complement system.
Nat Rev Drug Discov, [Epub ahead of print]. [PMID:31819218]

7. Xu B. (2023)
Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria.
Clin Exp Med, 23 (3): 717-726. [PMID:35441351]

8. (2024)
Pegcetacoplan versus Eculizumab in Paroxysmal Nocturnal Hemoglobinuria.
N Engl J Med, 390 (11): 1060. [PMID:38478008]