{"entity":{"id":"tovorafenib","kind":"drug","name":"Tovorafenib","aka":[],"tldr":"Tovorafenib is a pill for the most common childhood brain tumour, low-grade glioma driven by BRAF changes, approved in 2024.","summary":"FDA accelerated approval 23 April 2024 for relapsed/refractory paediatric low-grade glioma (age ≥6 months) with BRAF fusion/rearrangement or V600 mutation (FIREFLY-1: ORR ~51% by RANO-HGG). European conditional approval April 2026 (Ipsen, regardless of BRAF alteration type). Type II RAF inhibitor active against KIAA1549-BRAF fusions where type I inhibitors cause paradoxical activation. Phase 3 LOGGIPY-2 in first line versus chemotherapy. Day One Biopharmaceuticals.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA approval","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tovorafenib-patients-relapsed-or-refractory-braf-altered-pediatric"}],"tags":[],"related":["braf-fusion"],"cancers":["glioblastoma","paediatric-low-grade-glioma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":[],"companies":["day-one-biopharmaceuticals","ipsen"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct05566795"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ojemda","modality":"Small-molecule type II RAF inhibitor","mechanism":"Pan-RAF type II inhibitor binding the DFG-out conformation; blocks monomeric and dimeric BRAF signalling.","approvals":[{"region":"US","year":2024,"indication":"Relapsed/refractory BRAF-altered paediatric low-grade glioma (accelerated)"},{"region":"EU","year":2026,"indication":"Relapsed/refractory paediatric low-grade glioma (conditional)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-04-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.4 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tovorafenib-patients-relapsed-or-refractory-braf-altered-pediatric","indication":"Patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation."}]},"route":"/drugs/tovorafenib/","neighbours":{"biomarker":[{"id":"braf-class-ii-iii","kind":"biomarker","name":"BRAF class II and class III mutations (non-V600)","route":"/biomarkers/braf-class-ii-iii/"},{"id":"braf-fusion","kind":"biomarker","name":"BRAF fusion or rearrangement","route":"/biomarkers/braf-fusion/"}],"cancer":[{"id":"brain-tumours","kind":"cancer","name":"Brain and spinal cord tumours (all types)","route":"/cancers/brain-tumours/"},{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"glioblastoma","kind":"cancer","name":"Glioma & glioblastoma","route":"/cancers/glioblastoma/"},{"id":"optic-pathway-glioma","kind":"cancer","name":"Optic pathway glioma","route":"/cancers/optic-pathway-glioma/"},{"id":"paediatric-low-grade-glioma","kind":"cancer","name":"Paediatric low-grade glioma","route":"/cancers/paediatric-low-grade-glioma/"}],"technology":[{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"target":[{"id":"braf","kind":"target","name":"BRAF","route":"/targets/braf/"}],"company":[{"id":"day-one-biopharmaceuticals","kind":"company","name":"Day One Biopharmaceuticals","route":"/companies/day-one-biopharmaceuticals/"},{"id":"ipsen","kind":"company","name":"Ipsen","route":"/companies/ipsen/"}],"pathway":[{"id":"glioma-signalling","kind":"pathway","name":"Glioma (KEGG map)","route":"/pathways/glioma-signalling/"},{"id":"ras-mapk","kind":"pathway","name":"RAS / RAF / MEK / ERK (MAPK)","route":"/pathways/ras-mapk/"}],"trial":[{"id":"nct05566795","kind":"trial","name":"DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)","route":"/trials/nct05566795/"},{"id":"firefly-1","kind":"trial","name":"FIREFLY-1","route":"/trials/firefly-1/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-brain-delivery","kind":"bottleneck","name":"The brain: barrier and sanctuary","route":"/bottlenecks/b-brain-delivery/"}],"idea":[{"id":"idea-bio2-paediatric-first-development","kind":"idea","name":"Develop drugs in children first when the target is a children's target","route":"/ideas/idea-bio2-paediatric-first-development/"}],"term":[{"id":"race-for-children-act","kind":"term","name":"RACE for Children Act","route":"/terms/race-for-children-act/"}],"roadmap":[{"id":"paediatric-oncology-roadmap","kind":"roadmap","name":"Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first","route":"/roadmaps/paediatric-oncology-roadmap/"}]}}