Day One Biopharmaceuticals to file NDA for paediatric brain cancer treatment before second half of 2023
pharmafile | January 10, 2023 | News story | Research and Development |
Day One Biopharmaceuticals is set to ask the FDA for approval for tovorafenib before the summer of 2023. Tovorafenib is Day One’s paediatric brain cancer drug which has appeared positive in various trials.
The New Drug Application (NDA) is likely to include top-line data from the drug’s phase 2 trial, which demonstrated that 64% of 69 patients responded to the therapy. Most patients stayed on the drug for an average of 8.4 months with 77% of the trial patients still on the drug at the 28 September 2022 data cutoff point. The 64% statistic remains consistent with the smaller dataset of 25% patients assessed last year.
The FIRE-FLY-1 study included patients aged from 6 months to 25 years, who received tovorafenib once per week. The patients all had recurrent or progressive paediatric low-grade glioma, the most common brain cancer in children, according to Day One.
Day One has shared that three patients responded fully to the drug, while 41 responded partially – although 10 of this 41 were unconfirmed. The drug was generally well-tolerated, with a few side effects being mentioned. The most common side effect was a change in hair colour, although others included increasing creatine phosphokinase, anemia, fatigue and maculopapular rashes.
Jeremy Bender, Day One’s CEO, commented: “We look forward to continuing our discussions with regulatory authorities with the hope of bringing this therapy to children in need of new options as soon as possible.”