Day One Announces FDA Acceptance of NDA and Priority Review for Tovorafenib in Relapsed or Progressive Pediatric Low-Grade Glioma (pLGG)

Day One Biopharmaceuticals, a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for tovorafenib as a monotherapy in relapsed or progressive pediatric low-grade glioma (pLGG). The FDA has granted priority review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024. The FDA is not currently planning to hold an advisory committee meeting to discuss the application.

pLGG is the most common brain tumor diagnosed in children, with patients suffering profound tumor and treatment-associated morbidities that can impact their life trajectory. For the vast majority of patients in the relapsed setting, there is no standard of care and no approved therapy.

“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 alterations have relapsed or progressed,” said Jeremy Bender, Ph.D., chief executive officer of Day One. “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.”

The NDA is based on results from the open-label, pivotal Phase 2 trial evaluating tovorafenib as a once-weekly monotherapy in patients aged 6 months to 25 years with relapsed or progressive pLGG.
Updated data was recently disclosed when the Company announced the completion of its rolling NDA submission on September 11, 2023. New, detailed data is expected to be presented at an upcoming medical conference.

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, the Company may receive a voucher if it receives an approval for an eligible indication, which can be redeemed to obtain priority review for a subsequent marketing application for a different product candidate.

Tovorafenib is an investigational therapy that is not approved for commercial use in any country.