Alpelisib: First Global Approval

Drugs. 2019 Jul;79(11):1249-1253. doi: 10.1007/s40265-019-01161-6.

Abstract

Alpelisib (Piqray™)-an orally available phosphatidylinositol 3-kinase (PI3K) inhibitor with specific activity against PI3K alpha (PI3Kα)-is being developed by Novartis for the treatment of breast cancer. Alpelisib has demonstrated efficacy in combination with fulvestrant as treatment for hormone receptor (HR)-positive, human epidermal growth factor receptor-2 (HER2)-negative breast cancer in patients with a PIK3CA mutation and was recently approved for this indication in the USA. This article summarizes the milestones in the development of alpelisib leading to this first approval.

Publication types

  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / metabolism
  • Class I Phosphatidylinositol 3-Kinases / antagonists & inhibitors*
  • Class I Phosphatidylinositol 3-Kinases / genetics
  • Drug Approval
  • Female
  • Fulvestrant / administration & dosage
  • Humans
  • Male
  • Mutation
  • Protein Kinase Inhibitors / administration & dosage*
  • Receptor, ErbB-2 / metabolism
  • Thiazoles / administration & dosage*
  • United States
  • United States Food and Drug Administration

Substances

  • Protein Kinase Inhibitors
  • Thiazoles
  • Alpelisib
  • Fulvestrant
  • Class I Phosphatidylinositol 3-Kinases
  • PIK3CA protein, human
  • ERBB2 protein, human
  • Receptor, ErbB-2