Day One Biopharmaceuticals said Tuesday the Food and Drug Administration gave accelerated approval to its targeted pill for one of the most common forms of childhood brain tumors, pediatric low-grade glioma.
The weekly pill, previously known as tovorafenib, will be marketed as Ojemda. These tumors generally regress when patients become adults, but toxic treatments are often needed to keep them at bay until then. The hope is that Ojemda can spare some children from, for example, radiation, which can devastate tumors but also healthy tissue throughout the brain and bring lifelong consequences.
According to the most recent analysis of a Phase 2 trial, Ojemda shrank tumors in 51% of the 77 patients who received the drug, with responses lasting a median of 13.8 months. (Day One previously reported higher response figures, using different criteria.). That included a 49% response rate in patients who had already relapsed on a similar targeted drug. The most common side effects were changes in hair color, rashes, and fatigue.