Day One’s Ojemda Gets FDA Accelerated Approval for Pediatric Brain Tumor

FDA Exterior/iStock, Grandbrothers

Pictured: Facade of the FDA's office in Maryland/iStock, Grandbrothers

Day One Biopharmaceuticals on Tuesday announced that it has secured the FDA’s accelerated approval for tovorafenib, which will be marketed under the brand name Ojemda, for the treatment of relapsed or refractory pediatric low-grade glioma in patients aged six months and older.

According to Ojemda’s label, the oral kinase inhibitor is indicated for patients with confirmed BRAF fusion or rearrangement alterations, or a BRAF V600 mutation. The drug comes in tablet and oral suspension formulations and should be dosed once a week.

Under the FDA’s accelerated pathway, Ojemda’s continued approval depends on the verification of its clinical benefit in a confirmatory study. To fulfill this requirement, Day One is running the Phase III FIREFLY-2/LOGGIC trial, a randomized study comparing Ojemda versus chemotherapy in the front-line setting of pediatric low-grade glioma (pLGG).

The trial, which will include patients from six months through 25 years of age, is currently enrolling at various sites across the U.S., Canada, Asia, Australia and Europe.

Day One CEO Jeremy Bender in a statement said that Ojemda’s approval “ushers in a new day” for children with relapsed or refractory pLGG, adding that the drug will help address this “serious and life-threatening disease of adolescence.”

“Ojemda is the first and only FDA-approved medicine for children with BRAF fusions or rearrangements, which are the most common molecular alteration in pLGG,” Bender added. Ojemda is also the only systemic treatment option for pLGG that allows for a once-weekly treatment schedule, according to Day One.

Designed to be able to penetrate the blood-brain barrier, Ojemda is a type II RAF kinase blocker that works by binding a key player in the MAPK signaling cascade, disrupting the tumor cells’ unchecked growth and proliferation and triggering cell death.

Day One backed Ojemda’s regulatory bid with data from the open-label Phase II FIREFLY-1 study, which enrolled 137 patients across two study arms. The first consisted of 77 patients and showed a best overall response rate of 51%, which included 28% partial responses. Median duration of response was 13.8 months as of the data cut-off in June 2023.

The second arm included 60 patients and provided additional safety information. Overall, in 137 patients, Ojemda demonstrated a favorable safety and tolerability profile with most of its adverse events being grade 1 or grade 2 in severity. The most common side effects included rash, fatigue, viral infection, headache and fever.

Ojemda was developed in partnership with XOMA Corporation under a March 2021 partnership, when the biotech royalty aggregator invested $13.5 million upfront.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Back to news