Pralsetinib: First Approval

Drugs. 2020 Nov;80(17):1865-1870. doi: 10.1007/s40265-020-01427-4.

Abstract

Pralsetinib (GAVRETO™, Blueprint Medicines Corporation) is a selective rearranged during transfection (RET) inhibitor being developed for the treatment of various solid tumours. RET is a well described proto-oncogene present in multiple cancers including non-small cell lung cancer (NSCLC), papillary thyroid cancer, and medullary thyroid carcinoma (MTC). Pralsetinib was recently granted accelerated approval for the treatment of metastatic RET fusion-positive NSCLC in the USA and is under regulatory review in the USA for RET fusion-positive thyroid cancer and RET mutation-positive MTC; pralsetinib is under regulatory review in the EU for RET fusion-positive NSCLC. This article summarizes the milestones in the development of pralsetinib leading to this first approval.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Carcinoma, Neuroendocrine / drug therapy
  • Carcinoma, Neuroendocrine / genetics
  • Carcinoma, Neuroendocrine / mortality
  • Carcinoma, Neuroendocrine / pathology
  • Carcinoma, Non-Small-Cell Lung / drug therapy
  • Carcinoma, Non-Small-Cell Lung / genetics
  • Carcinoma, Non-Small-Cell Lung / mortality
  • Carcinoma, Non-Small-Cell Lung / pathology
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Clinical Trials, Phase III as Topic
  • Drug Approval*
  • Humans
  • Lung Neoplasms / drug therapy
  • Lung Neoplasms / genetics
  • Lung Neoplasms / mortality
  • Lung Neoplasms / pathology
  • Multicenter Studies as Topic
  • Oncogene Proteins, Fusion / antagonists & inhibitors*
  • Oncogene Proteins, Fusion / genetics
  • Progression-Free Survival
  • Proto-Oncogene Mas
  • Proto-Oncogene Proteins c-ret / antagonists & inhibitors*
  • Proto-Oncogene Proteins c-ret / genetics
  • Pyrazoles / pharmacology
  • Pyrazoles / therapeutic use*
  • Pyridines / pharmacology
  • Pyridines / therapeutic use*
  • Pyrimidines / pharmacology
  • Pyrimidines / therapeutic use*
  • Randomized Controlled Trials as Topic
  • Thyroid Cancer, Papillary / drug therapy
  • Thyroid Cancer, Papillary / genetics
  • Thyroid Cancer, Papillary / mortality
  • Thyroid Cancer, Papillary / pathology
  • Thyroid Neoplasms / drug therapy
  • Thyroid Neoplasms / genetics
  • Thyroid Neoplasms / mortality
  • Thyroid Neoplasms / pathology
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence

Substances

  • Antineoplastic Agents
  • MAS1 protein, human
  • Oncogene Proteins, Fusion
  • Proto-Oncogene Mas
  • Pyrazoles
  • Pyridines
  • Pyrimidines
  • pralsetinib
  • Proto-Oncogene Proteins c-ret
  • RET protein, human

Supplementary concepts

  • Thyroid cancer, medullary