FDA

Day One Biopharmaceuticals drug Ojemda is now FDA-approved for advanced pediatric low-grade glioma, the most common type of brain cancer in children. The regulatory decision for Ojemda covers a broader swath of patients than a drug combination from Novartis approved for treating this childhood cancer....

The US Food and Drug Administration (FDA) finalized guidance on drug development programs for rare diseases, eliminating a section on natural history and providing additional considerations related to patient and caregiver input and pediatric populations....

New clinical treatments for diseases are conceived in a laboratory, then tested on animals or through simulations, and finally studied on human beings if deemed promising enough. Moving from point to point in this cycle is rarely a straight line, and costly and time-consuming trial-and-...