The FDA granted accelerated approval to Day One Biopharmaceuticals’ pediatric brain tumor treatment.
The regulator on Tuesday approved tovorafenib, taking place more than five years after Day One was founded with a goal to bring targeted therapies to children, a population that the drug industry has largely treated as an afterthought.
Tovorafenib, to be marketed as Ojemda, is approved for children 6 months of age and older with low-grade glioma that has stopped responding to or returned after previous treatment. Ojemda, once a Takeda drug candidate, is a type of targeted treatment known as a BRAF inhibitor. To qualify for treatment, patients need to have tumors with a BRAF fusion or rearrangement, or BRAF V600E mutation.
In a securities filing Wednesday, Day One said a 28-day supply of Ojemda will be priced at $33,916.
When the company launched in 2018, Day One’s plan was to develop a targeted treatment first in a pediatric population and then do so in adults. While laws in the US and Europe mandate that companies run clinical trials of adult cancer drugs in kids, few treatments are developed specifically for children.
“It’s really to address the innovation gap between children and adults who are living with cancer and to do so with a business model that is both creative and focused on sustainability,” said CEO Jeremy Bender, who joined the company in 2020.
Low-grade gliomas, which are slow-growing, are the most common type of pediatric brain cancer. They are generally treatable in children with good survival odds, but patients might experience vision changes, balance issues and headaches, among other symptoms.
These tumors are typically treated with older therapies. Children diagnosed with low-grade glioma typically first receive surgery if they are eligible. Roughly one-third have their tumors completely resected; for the rest of the patients, the remaining options are chemotherapy (and possibly several rounds of it) and then radiation, which can come with both short- and long-term side effects.
Minor response
Ojemda is the second targeted treatment approved for children with low-grade glioma. The FDA last year approved Novartis’ combination of the BRAF inhibitor Tafinlar plus Mekinist for patients 1 year of age and older with a BRAF V600E mutation. That’s roughly 15% to 20% of pediatric patients with BRAF changes.
Ojemda’s approval is the first for a systemic therapy for patients with BRAF fusion or rearrangements, according to the FDA.
The accelerated approval was granted based on the results of a Phase 2 study, FIREFLY-1, in which 51% of patients saw their tumors shrink at least 25% after receiving Ojemda. In 76 patients, 28 saw partial responses (meaning their tumors shrank at least 50%) and 11 saw minor responses — their tumors shrank between 25% and 49%.
Sam BlackmanThe treatment is offered as both a tablet and oral suspension. The drug comes with a safety warning and precaution that it can slow height growth. Day One’s R&D head Sam Blackman said that the effect appears reversible and “patients appear to catch up when they come off the drug.”
According to Blackman, it’s the first time the FDA has included minor responses in the response criteria of a drug label. “I would argue — as we argued to the FDA — that if you have a tumor that shrinks by 48%, that’s a big deal for relapsed low-grade glioma,” Blackman said. “It’s a big deal for that child because things are growing inside your brain. That’s not a place where things should grow.”
Day One said it plans to use a Phase 3 trial called FIREFLY-2 as the confirmatory study for the accelerated approval. The company also received a rare pediatric disease priority review voucher from the FDA, which it plans to sell. Typically companies can net around $100 million from selling the vouchers. The program that grants the vouchers is currently at risk of not being renewed by lawmakers.
“We now have shown that you can develop drugs intentionally for children at the speed of biotech,” Blackman said.
The company is also developing tovorafenib as part of a combination for adults.
Day One’s stock $DAWN jumped 10% Tuesday afternoon after the news was announced.
Editor’s note: This story was updated to include the price of the drug.