Pegcetacoplan

Review

Pegcetacoplan

No authors listed

In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.

2024 Jul 20.

PMID: 39083628
Bookshelf ID: NBK605496

Free Books & Documents

Review

Pegcetacoplan

No authors listed.

Free Books & Documents

In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.

2024 Jul 20.

PMID: 39083628
Bookshelf ID: NBK605496

Excerpt

Pegcetacoplan is a small peptide inhibitor of complement that is used to treat adults with paroxysmal nocturnal hemoglobinuria. Serum aminotransferase elevations occur in a proportion of patients treated pegcetacoplan, but episodes of clinically apparent liver injury with jaundice have not been reported with its use.

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1. Zimmerman HJ. Hepatotoxicity: the adverse effects of drugs and other chemicals on the liver. 2nd ed. Philadelphia: Lippincott, 1999.(Review of hepatotoxicity published in 1999 before the availability of complement inhibitors).
1. Wellstein A, Giaccone G, Atkins MB, Sausville EA. Pathway targeted therapies: monoclonal antibodies, protein kinase inhibitors, and various small molecules. In, Brunton LL Hilal-Dandan R, Knollman BC, eds. Goodman & Gilman's the pharmacological basis of therapeutics. 13th ed. New York: McGraw-Hill, 2018, pp. 1203-36.(Textbook of pharmacology and therapeutics).
1. FDA. Integrated Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215014Orig1s000I...(FDA website with initial multidiscipline clinical review of the safety and efficacy of pegcetacoplan as therapy of PNH, describes the adverse events observed in a pooled safety population which were largely due to episodes of hemolysis, thromboses, and infections, and while elevations in liver enzymes were not infrequent in some studies, they were attributed to hemolysis, thromboses and infections rather than liver injury due to pegcetacoplan).
1. Hillmen P, Szer J, Weitz I, Röth A, Höchsmann B, Panse J, Usuki K, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384:1028-1037.(Among 80 adults with PNH and incomplete response to eculizumab [hemoglobin less than 10.5 g/dL] treated with pegcetacoplan vs eculizumab, mean hemoglobin levels improved only with pegcetacoplan [rising to 11.5 vs 8.6 g/dL], fewer required transfusions [15% vs 85%], and fatigue and quality of life improved, while adverse event rates included infusion site reactions [37% vs 3%], diarrhea [22% vs 3%], breakthrough hemolysis [10% vs 23%], headache [7% vs 23%], fatigue [5% vs 15%]; no mention of ALT elevations or hepatic adverse events). - PubMed
1. Hoy SM. Pegcetacoplan: first approval. Drugs. 2021;81:1423-1430.(Review of the mechanism of action, history of development, pharmacology, clinical efficacy, and safety of pegcetacoplan in PNH, age-related macular degeneration, C3 glomerulopathy, and autoimmune hemolytic anemia; no mention of ALT elevations or hepatic adverse events). - PubMed

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[1] Zimmerman HJ. Hepatotoxicity: the adverse effects of drugs and other chemicals on the liver. 2nd ed. Philadelphia: Lippincott, 1999.(Review of hepatotoxicity published in 1999 before the availability of complement inhibitors).

[2] Zimmerman HJ. Hepatotoxicity: the adverse effects of drugs and other chemicals on the liver. 2nd ed. Philadelphia: Lippincott, 1999.(Review of hepatotoxicity published in 1999 before the availability of complement inhibitors).

[3] Wellstein A, Giaccone G, Atkins MB, Sausville EA. Pathway targeted therapies: monoclonal antibodies, protein kinase inhibitors, and various small molecules. In, Brunton LL Hilal-Dandan R, Knollman BC, eds. Goodman & Gilman's the pharmacological basis of therapeutics. 13th ed. New York: McGraw-Hill, 2018, pp. 1203-36.(Textbook of pharmacology and therapeutics).

[4] Wellstein A, Giaccone G, Atkins MB, Sausville EA. Pathway targeted therapies: monoclonal antibodies, protein kinase inhibitors, and various small molecules. In, Brunton LL Hilal-Dandan R, Knollman BC, eds. Goodman & Gilman's the pharmacological basis of therapeutics. 13th ed. New York: McGraw-Hill, 2018, pp. 1203-36.(Textbook of pharmacology and therapeutics).

[5] FDA. Integrated Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215014Orig1s000I...(FDA website with initial multidiscipline clinical review of the safety and efficacy of pegcetacoplan as therapy of PNH, describes the adverse events observed in a pooled safety population which were largely due to episodes of hemolysis, thromboses, and infections, and while elevations in liver enzymes were not infrequent in some studies, they were attributed to hemolysis, thromboses and infections rather than liver injury due to pegcetacoplan).

[6] FDA. Integrated Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215014Orig1s000I...(FDA website with initial multidiscipline clinical review of the safety and efficacy of pegcetacoplan as therapy of PNH, describes the adverse events observed in a pooled safety population which were largely due to episodes of hemolysis, thromboses, and infections, and while elevations in liver enzymes were not infrequent in some studies, they were attributed to hemolysis, thromboses and infections rather than liver injury due to pegcetacoplan).

[7] Hillmen P, Szer J, Weitz I, Röth A, Höchsmann B, Panse J, Usuki K, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384:1028-1037.(Among 80 adults with PNH and incomplete response to eculizumab [hemoglobin less than 10.5 g/dL] treated with pegcetacoplan vs eculizumab, mean hemoglobin levels improved only with pegcetacoplan [rising to 11.5 vs 8.6 g/dL], fewer required transfusions [15% vs 85%], and fatigue and quality of life improved, while adverse event rates included infusion site reactions [37% vs 3%], diarrhea [22% vs 3%], breakthrough hemolysis [10% vs 23%], headache [7% vs 23%], fatigue [5% vs 15%]; no mention of ALT elevations or hepatic adverse events). - PubMed

[8] Hillmen P, Szer J, Weitz I, Röth A, Höchsmann B, Panse J, Usuki K, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384:1028-1037.(Among 80 adults with PNH and incomplete response to eculizumab [hemoglobin less than 10.5 g/dL] treated with pegcetacoplan vs eculizumab, mean hemoglobin levels improved only with pegcetacoplan [rising to 11.5 vs 8.6 g/dL], fewer required transfusions [15% vs 85%], and fatigue and quality of life improved, while adverse event rates included infusion site reactions [37% vs 3%], diarrhea [22% vs 3%], breakthrough hemolysis [10% vs 23%], headache [7% vs 23%], fatigue [5% vs 15%]; no mention of ALT elevations or hepatic adverse events). - PubMed

[9] Hoy SM. Pegcetacoplan: first approval. Drugs. 2021;81:1423-1430.(Review of the mechanism of action, history of development, pharmacology, clinical efficacy, and safety of pegcetacoplan in PNH, age-related macular degeneration, C3 glomerulopathy, and autoimmune hemolytic anemia; no mention of ALT elevations or hepatic adverse events). - PubMed

[10] Hoy SM. Pegcetacoplan: first approval. Drugs. 2021;81:1423-1430.(Review of the mechanism of action, history of development, pharmacology, clinical efficacy, and safety of pegcetacoplan in PNH, age-related macular degeneration, C3 glomerulopathy, and autoimmune hemolytic anemia; no mention of ALT elevations or hepatic adverse events). - PubMed

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Pegcetacoplan

Pegcetacoplan

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