Tovorafenib for treating relapsed or refractory paediatric low-grade glioma in patients aged 6 months and older

Tovorafenib is currently in clinical development for the treatment of relapsed or refractory paediatric low-grade glioma (LGG) in patients aged 6 months and older. Gliomas are cancerous brain tumours that start in glial cells, which are the supporting cells of the brain and the spinal cord. Paediatric LGGs (pLGG) are the most frequent brain tumours diagnosed in children and young adults, accounting for approximately 40% of all reported cases. Like most brain tumours the cause of LGG is unknown in most cases, however, gene alterations are responsible for almost all pLGGs. Some symptoms include nausea and vomiting, lethargy and irritability, headaches, clumsiness, and seizures. Standard treatments for gliomas, like surgery, radiation, and chemotherapy, often lead to poor post-treatment quality of life, poor survival rates and long-term side effects, hence the need for more effective therapies.