Type II RAF inhibitor tovorafenib for the treatment of pediatric low-grade glioma.

Expert Rev Clin Pharmacol

The First Affiliated Hospital, Hunan Provincial Clinical Medical Research Center for Drug Evaluation of Major Chronic Diseases, Hengyang Medical School, University of South China, Hengyang, Hunan, China.

Published: November 2024

AI Article Synopsis

  • Pediatric low-grade glioma (pLGG) is the most common type of brain tumor in children and is now treated as a chronic disease; recently, the FDA approved a new drug, tovorafenib, for patients aged 6 months and older with specific genetic mutations in pLGG.
  • The article reviews tovorafenib's pharmacological properties, effectiveness, and safety through a systematic search of relevant publications.
  • Tovorafenib is the first FDA-approved treatment for pediatric patients with common BRAF mutations in pLGG, exhibiting good CNS penetration and demonstrating positive clinical results in trials with minimal side effects.

Article Abstract

Introduction: Pediatric low-grade glioma (pLGG) is the most prevalent childhood brain tumor group, currently regarded as a chronic disease. On 23 April 2024, the U.S. FDA approved a new type II RAF inhibitor, tovorafenib (OJEMDA), previously known as DAY101, for the treatment of patients aged 6 months and older with relapsed or refractory (R/R) pLGG harboring a BRAF fusion or rearrangement, or BRAF V600E mutation.

Areas Covered: This article aims to review the pharmacological properties of tovorafenib and evaluate its efficacy and safety in the treatment of R/R pLGG. We conducted a systematic search of PubMed and Web of Science databases for English-language publications related to tovorafenib, including journal articles and conference abstracts, up through 20 August 2024.

Expert Opinion: As the first and only FDA-approved medicine for children with BRAF fusions or rearrangements, which are the most common molecular alteration in pLGG, tovorafenib shows superior central nervous system penetration without the paradoxical activation of the MAPK pathway reported for type I BRAF inhibitors. Phase 1 and the pivotal phase 2 trials have demonstrated that tovorafenib monotherapy is generally well-tolerated and exhibits encouraging signs of meaningful, rapid and sustained clinical activity in children and young adults with BRAF-altered pLGG.

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Source
http://dx.doi.org/10.1080/17512433.2024.2418405DOI Listing

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