The European Commission has granted conditional approval to tovorafenib (Ojemda) for children aged 6 months and older with relapsed or refractory pediatric low-grade glioma (pLGG) carrying BRAF alterations. This is the first systemic therapy specifically approved in the EU for BRAF-rearranged pLGG.
The decision is based on results from the FIREFLY-1 trial, where the drug showed strong effectiveness in patients who had already received prior treatment. Response rates reached up to 71%, with many patients experiencing long-lasting tumor control. Some children who completed treatment remained stable without needing further therapy for over a year, and those who relapsed could still respond to the drug again.
Side effects were considered manageable, though many patients experienced issues such as slowed growth, anemia, fatigue, and skin reactions. About 13% stopped treatment due to side effects.
This approval follows earlier authorization by the U.S. Food and Drug Administration and offers a new targeted treatment option for this challenging pediatric brain cancer.