FDA gives nod to Day One’s drug for common childhood brain tumor

Day One Biopharmaceuticals said Tuesday the Food and Drug Administration approved its pill for one of the most common forms of childhood brain tumors, called pediatric low-grade glioma.

The authorization is particularly notable because industry has historically developed few drugs for children with cancer, focusing instead on adults and often only years later testing their molecules in younger patients. Day One was established by Sam Blackman, a pediatric oncologist and longtime drug executive, and venture capitalist Julie Grant to try to find a model to develop drugs for children. 

The drug, previously known as tovorafenib, will be marketed as Ojemda. Day One has not yet disclosed a price.

Ojemda targets a protein called BRAF, which is the most common mutation driving such tumors, and is only indicated only for patients with a confirmed mutation.