The US Food and Drug Administration (FDA) has accepted Day One Biopharmaceuticals’ new drug application (NDA) and priority review for tovorafenib as a single agent in relapsed or progressive paediatric low-grade glioma (pLGG).

pLGG is the most detected brain tumour among children.

The NDA is based on data from the pivotal, open-label Phase II study assessing tovorafenib as a once-weekly monotherapy to treat patients aged between six and 25 years with relapsed or progressive pLGG.

Tovorafenib is an investigational, oral, brain-penetrant, type II RAF kinase inhibitor that targets a crucial enzyme in the mitogen-activated protein kinase (MAPK) signalling pathway.

It is currently under assessment in primary brain tumours or brain metastases of solid tumours.

Tovorafenib has been evaluated in 325 patients and is currently under assessment in two pivotal studies for pLGG.

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It is also being assessed alone or as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumours with MAPK pathway aberrations.

Day One CEO Jeremy Bender stated: “We are pleased to be one step closer to achieving our mission of bringing a novel targeted therapy to children whose low-grade gliomas with BRAF [v-raf murine sarcoma viral oncogene homolog B1] alterations have relapsed or progressed.

“We are grateful to the patients and their caregivers who participated in the FIREFLY-1 trial and look forward to continuing to collaborate with the FDA as we prepare to make this treatment more broadly available to those who need it.”

This content was updated on 25 January 2024