The FDA approved a new treatment for a rare form of brain cancer whose tumors have a mutation that drives the disease.
Tuesday’s approval of the drug, known as vorasidenib, was granted to Servier Pharmaceuticals for patients 12 and older with a grade 2 astrocytoma or oligodendroglioma that has an IDH1 or IDH2 mutation.
Servier said the price of Voranigo is $39,881 per month. Patients are eligible for the drug after surgery for their brain tumor.
The slow-growing brain tumors generally impact relatively young adults from their 20s to 50s. More than 80% of grade 2 gliomas bear an IDH mutation, and the FDA said this was the first approved systemic drug for this subset of patients. Servier estimates that roughly 3,000 patients are diagnosed with an IDH-mutant diffuse glioma every year in the US, while another 20,000 patients currently live with the disease.
In clinical trials, the drug delivered results that physicians described as “striking.” Compared to placebo, the treatment staved off cancer progression for a median 16.6 months longer — well over a year of additional time.
Servier acquired the drug in 2022 from Agios, which was making a pivot to rare disease. Through an agreement with Royalty Pharma — a niche company that buys drug royalties — Agios expects to receive a total of $1.1 billion in payments from the FDA approval, it said earlier this month with its financial results.
Susan Pandya, Servier’s head of cancer metabolism who joined from Agios, said that the drug, to be sold as Voranigo, “can penetrate the blood brain barrier, which is one of the biggest challenges for brain tumor research, and one that, unfortunately, the field has not been able to move the needle on for decades.”
“This will represent a major advancement in nearly 25 years in this disease space,” she said in an interview with Endpoints News ahead of the approval.
The approval comes roughly two weeks earlier than expected, as the FDA had a deadline of Aug. 20 to make its decision.
Editor’s note: This story has been updated with details on Agios’ payout and a comment from Susan Pandya, as well as Voranigo’s list price.