Day One Biopharmaceuticals Receives FDA Approval for Ojemda in pLGG
Day One Biopharmaceuticals, Inc. (NASDAQ: DAWN) has announced that its drug Ojemda (tovorafenib) has received an accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory pediatric Low-Grade Glioma (pLGG) harboring a BRAF fusion, rearrangement, or BRAF V600 mutation.
pLGG is the most common brain tumor diagnosed in children. Up to 75% of children with pLGG have a BRAF alteration, which is a genetic mutation that can lead to uncontrolled cell growth. Until now, there have been no drugs approved for patients with pLGG driven by BRAF alterations.
Ojemda is an orally administered, once-weekly, central nervous system (CNS) penetrant pan-RAF inhibitor. In a Phase 2 clinical study, 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).
Market Opportunity
The prevalence of systemically treated BRAF-altered pLGG patients in the U.S. is estimated to be approximately 26,000. The estimated recurrent or progressive total addressable patient population per annum is approximately 2,000-3,000.
Ojemda has a wider label than other drugs approved for pLGG, which means that it can be used to treat a larger population of patients. Day One estimates that Ojemda has a peak revenue opportunity of approximately $500-$600 million per year.
Analyst Commentary
Analysts are generally positive on the news of Ojemda’s approval. They believe that the drug has the potential to be a blockbuster drug and that Day One is a well-positioned company to capitalize on this opportunity.
Conclusion
The FDA approval of Ojemda is a major milestone for Day One Biopharmaceuticals and for patients with pLGG. Ojemda is the first drug approved for this type of cancer and has the potential to significantly improve the lives of these patients.
