The company is targeting around 200 centers that treat the vast majority of the few thousand pediatric low-grade glioma patients who are candidates for the RAF-targeting drug.

Glioblastoma carries a grim prognosis. The biopharma industry is trying to improve patient outcomes, with 120 drugs in the pipeline and two anticipated approvals this year.

User fee goal dates in April include four novel agents with breakthrough therapy designations, including Pfizer’s hemophilia B gene therapy and Immunity Bio’s IL-15 superagonist complex for bladder cancer.

More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.