Day One Biopharma’s Ojemda: A Long-Awaited Approval for pLGG Patients

Day One Biopharmaceuticals, Inc. (NASDAQ: DAWN) has received an accelerated approval from the FDA for its drug Ojemda (tovorafenib) for the treatment of relapsed or refractory pediatric Low-Grade Glioma (pLGG) harboring a BRAF fusion, rearrangement, or BRAF V600 mutation. Ojemda is the first drug approved for patients with pLGG driven by BRAF alterations, which account for up to 75 percent of children with this type of brain cancer. The approval was based on results from Day One’s pivotal, Phase 2 open-label FIREFLY-1 clinical study, which enrolled 137 patients. The study showed that Ojemda achieved an Overall Response Rate (ORR) of 51%, with 28% Partial Responses (PRs) and 11% Minor Responses (MRs). The ORR was 52% among the 64 patients with BRAF fusions or rearrangements and 50% for the 12 patients with a BRAF V600 mutation. The median duration of response was 13.8 months. The most common treatment-emergent adverse events (TEAEs) were anemia and elevated creatine phosphokinase (CPK). Day One is currently conducting a Phase 3 FIREFLY-2/LOGGIC randomized clinical trial evaluating tovorafenib as a potential front-line therapy compared to chemotherapy in patients aged 6 months to 25 years with pLGG.

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