FDA Grants Accelerated Approval to Day One’s Ojemda for Childhood Brain Tumor

Rare Daily Staff

The U.S. Food and Drug Administration has granted accelerated approval to Day One Biopharmaceuticals’ Ojemda for a type of rare, childhood brain tumor.

The accelerated approval, based on response rate and duration of response, is for the use of Ojemda for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. Day One received a rare pediatric disease priority review voucher from the FDA.

“Ojemda ushers in a new day for children living with relapsed or refractory pediatric low-grade glioma (pLGG),” said Jeremy Bender, CEO of Day One. “Moreover, 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.”

Pediatric low-grade glioma (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. BRAF is the most commonly altered gene in pLGG, with up to 75 percent of children having a BRAF alteration. In children with BRAF-altered pLGG, approximately 80 percent have BRAF fusions or rearrangements, while the remaining 20 percent have a V600 mutation. Until now, there had been no medicines approved for patients with pLGG driven by BRAF fusions.

Ojemda is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. It is the only systemic therapy for pLGG that offers once-weekly dosing, with or without food, as a tablet or oral suspension. The FDA granted Ojemda Breakthrough Therapy and Rare Pediatric Disease designations for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. The FDA also granted Ojemda Orphan Drug designation for the treatment of malignant glioma and the European Commission granted it Orphan Drug designation for the treatment of glioma.

FDA’s accelerated approval of Ojemda is based on data from the company’s pivotal open-label phase 2 FIREFLY-1 trial, which enrolled a total of 137 relapsed or refractory BRAF-altered pLGG patients across two study arms. Arm 1, which accrued 77 patients, was used for the efficacy analyses. Arm 2 provided additional safety data from an incremental 60 patients and was initiated to enable access to tovorafenib once Arm 1 had fully accrued.

The most common side effects were rash, hair color changes, tiredness, viral infection, vomiting, headache, fever, dry skin, constipation, nausea, acne and upper respiratory tract infection.

Day One is currently enrolling patients in the 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, which it believes will satisfy certain post-marketing requirements to the FDA. This study is currently enrolling patients in the United States, Canada, Europe, Australia, and Asia.

Ojemda will be available in the United States through specialty pharmacy partners Biologics and Onco360.

Photo: Jeremy Bender, CEO of Day One