iptacopan

{
  "id": "MLC-T-ONC-014566",
  "term": "iptacopan",
  "field": "Oncology and biomedicine",
  "definition": "An orally available, small-molecule inhibitor of complement factor B (FB) with potential immunomodulatory activity. Upon administration, iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb). This limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders such as paroxysmal nocturnal hemoglobinuria (PNH).",
  "synonyms": [
    "factor B inhibitor LNP023"
  ],
  "references": [
    "National Cancer Institute Thesaurus"
  ],
  "url": "https://mlchart.com/terminology/oncology/iptacopan/"
}