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Tovorafenib (DAY101)

RAF-altered pediatric low-grade glioma requiring first-line systemic therapy

Also known as TAK-580
Regulatory submission
Not announced
Launch
Not announced
Last updated
155 sources

Product information and disease descriptionProduct descriptionProduct information

Indication

5 evidence topics · 6 sources

Summary: labeled indications and first-line use

The US and EU labels restrict tovorafenib to relapsed or refractory disease (US) or disease that progressed after one or more prior systemic therapies (EU). Neither label includes first-line (treatment-naive) systemic therapy of pLGG, so first-line use in the US and EU is outside the approved indication until FIREFLY-2 results are reviewed. The Japanese indication statement issued with the September 2026 approval (low-grade glioma with a BRAF gene mutation or fusion gene) does not name a line of therapy, although the supporting data came from FIREFLY-1 in relapsed or progressive disease.

United States: labeled indication, relapsed or refractory pLGG

“OJEMDA is indicated for the treatment of 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.” (opens the source at this quote in a new tab)

“This indication is approved under accelerated approval based on response rate and duration of response” (opens the source at this quote in a new tab)

“Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).” (opens the source at this quote in a new tab)

European Union: labeled indication, pLGG progressed after prior systemic therapy

“Ojemda is indicated as monotherapy for the treatment of patients 6 months of age and older with paediatric low-grade glioma (LGG) harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation, who have progressed after one or more prior systemic therapies (for biomarkers-based patient selection, see section 4.2).” (opens the source at this quote in a new tab)

Japan: approved indication

“オジェンダ®（一般名：トボラフェニブ）について、「BRAF遺伝子変異又は融合遺伝子を有する低悪性度神経膠腫」を効能又は効果として、厚生労働省より製造販売承認を取得したことをお知らせいたします。” (opens the source at this quote in a new tab)

“The efficacy and safety of tovorafenib for relapsed or progressive low-grade glioma with BRAF V600 alterations or BRAF fusion gene-positive were evaluated in an overseas Phase II clinical study (FIREFLY-1 study).” (opens the source at this quote in a new tab)

First-line pLGG: indication under evaluation

“Tovorafenib is under evaluation as a therapy for patients aged less than 25 years with pLGG harboring BRAF fusion or rearrangement, or BRAF V600 mutation requiring front-line treatment (Phase III FIREFLY-2/LOGGIC).” (opens the source at this quote in a new tab)