Tagraxofusp: First Global Approval

Drugs. 2019 Apr;79(5):579-583. doi: 10.1007/s40265-019-01087-z.

Abstract

Tagraxofusp (tagraxofusp-erzs) [Elzonris™] is an intravenously administered CD123-directed cytotoxin (composed of human interleukin-3 and a truncated diphtheria toxin payload) that was developed by Stemline Therapeutics, Inc. for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). In December 2018, tagraxofusp received its first global approval in the USA for the treatment of BPDCN in adults and in paediatric patients aged 2 years and older. A centralized registration application for the use of tagraxofusp in patients with BPDCN is under review in the EU. This article summarizes the milestones in the development of tagraxofusp leading to its first global approval for the treatment of BPDCN.

Publication types

  • Review

MeSH terms

  • Administration, Intravenous
  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / pharmacokinetics*
  • Antineoplastic Agents / therapeutic use
  • Child
  • Child, Preschool
  • Diphtheria Toxin / administration & dosage
  • Diphtheria Toxin / adverse effects
  • Diphtheria Toxin / pharmacokinetics*
  • Diphtheria Toxin / therapeutic use
  • Drug Approval
  • Female
  • Humans
  • Interleukin-3 / administration & dosage
  • Interleukin-3 / adverse effects
  • Interleukin-3 / pharmacokinetics*
  • Interleukin-3 / therapeutic use
  • Interleukin-3 Receptor alpha Subunit / metabolism
  • Male
  • Middle Aged
  • Molecular Targeted Therapy / methods
  • Skin Neoplasms / drug therapy*
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration
  • Young Adult

Substances

  • Antineoplastic Agents
  • Diphtheria Toxin
  • IL3 protein, human
  • IL3RA protein, human
  • Interleukin-3
  • Interleukin-3 Receptor alpha Subunit