FDA Accepts Day One Biopharmaceuticals’ NDA for Pediatric Glioma Treatment
The FDA has accepted Day One Biopharmaceuticals’ (Nasdaq: DAWN) New Drug Application (NDA) for tovorafenib as a monotherapy in relapsed or progressive pediatric low-grade glioma (pLGG). The FDA has also granted priority review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
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.
Jeremy Bender, Ph.D., chief executive officer of Day One, said, “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. 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.”
Highlights
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.
FIREFLY-1 is evaluating tovorafenib as once-weekly monotherapy in patients aged 6 months to 25 years with relapsed or progressive pLGG harboring a known activating BRAF alteration.
The primary endpoint is overall response rate (ORR), defined as the proportion of patients with confirmed response based upon Response Asessment for Neuro-Oncology High Grade Glioma (RANO-HGG) criteria.
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.
Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children, accounting for 30% – 50% of all central nervous systems tumors.
About Day One Biopharmaceuticals
Day One Biopharmaceuticals is a clinical-stage biopharmaceutical Company that believes when it comes to pediatric cancer, we can do better. We put kids first and are developing targeted therapies that deliver to their needs. Day One was founded to address a critical unmet need: the dire lack of therapeutic development in pediatric cancer. The Company’s name was inspired by “The Day One Talk” that physicians have with patients and their families about an initial cancer diagnosis and treatment plan. Day One aims to re-envision cancer drug development and redefine what’s possible for all people living with cancer—regardless of age—starting from Day One.
Day One partners with leading clinical oncologists, families, and scientists to identify, acquire, and develop important emerging cancer treatments. The Company’s lead product candidate, tovorafenib, is an investigational, oral, brain-penetrant, highly-selective type II RAF kinase inhibitor. The Company’s pipeline also includes pimasertib, an investigational, oral, highly-selective small molecule inhibitor of mitogen‐activated protein kinases 1 and 2 (MEK-1/-2). Day One is based in Brisbane, California. For more information, please visit www.dayonebio.com or find the Company on LinkedIn or X/Twitter.
