Journal Article

Pexidartinib: First Approval

Lamb, Yvette N.

2019

Overview

Pexidartinib (TURALIO™) is an orally administered small molecule tyrosine kinase inhibitor with selective activity against the colony-stimulating factor 1 (CSF1) receptor, KIT proto-oncogene receptor tyrosine kinase (KIT) and FMS-like tyrosine kinase 3 harboring an internal tandem duplication mutation (FLT3-ITD). In August 2019, the US FDA approved pexidartinib capsules for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. This approval was based on positive results from the phase III ENLIVEN trial. Pexidartinib is being investigated in various malignancies as monotherapy or combination therapy. This article summarizes the milestones in the development of pexidartinib leading to its first approval for TGCT.

• Publisher: Springer International Publishing,Springer Nature B.V
• Subject: AdisInsight Report / Aminopyridines - administration & dosage / Aminopyridines - chemistry / Aminopyridines - pharmacology / Antineoplastic Agents - chemistry / Antineoplastic Agents - pharmacology / Colony-stimulating factor / Drug Approval / Drug dosages / Enzyme inhibitors / Flt3 protein / fms-Like Tyrosine Kinase 3 - antagonists & inhibitors / fms-Like Tyrosine Kinase 3 - metabolism / Gene expression / Giant Cell Tumor of Tendon Sheath - drug therapy / Giant Cell Tumor of Tendon Sheath - metabolism / Humans / Internal Medicine / Kinases / Macrophage colony-stimulating factor / Macrophage Colony-Stimulating Factor - antagonists & inhibitors / Macrophage Colony-Stimulating Factor - metabolism / Medicine / Medicine & Public Health / Morbidity / Mutation / Oral administration / Pharmacokinetics / Pharmacology/Toxicology / Pharmacotherapy / Protein Kinase Inhibitors - administration & dosage / Protein Kinase Inhibitors - chemistry / Protein Kinase Inhibitors - pharmacology / Protein-tyrosine kinase receptors / Proto-Oncogene Proteins c-kit - antagonists & inhibitors / Proto-Oncogene Proteins c-kit - metabolism / Pyrroles - administration & dosage / Pyrroles - chemistry / Pyrroles - pharmacology / Receptors / Surgery / Tumors / Tyrosine / United States / United States Food and Drug Administration