The FDA approved Servier Pharmaceuticals’ Voranigo as a treatment for two types of low-grade gliomas. The drug, which is projected to become a blockbuster product, comes from Servier’s $1.8 billion acquisition of Agios Pharmaceuticals’ oncology business.

By Frank Vinluan 

A Servier Pharmaceuticals drug designed to penetrate the brain to hit its targets is now FDA approved for treating two rare types of brain cancer.
The FDA approval announced Tuesday covers the treatment of adults and adolescents 12 and older who have Grade 2 astrocytoma or oligodendroglioma. The drug, vorasidenib, may be prescribed after surgery, as long as the cancers have mutations to the IDH1 or IDH2 enzymes the once-daily pill is designed to inhibit. The FDA said this regulatory decision marks the agency’s first for a systemic therapy for these cancers driven by either the IDH1 or IDH2 mutations. France-based Servier, which has its U.S. headquarters in Boston, will commercialize its new cancer drug under the brand name Voranigo.
Astrocytoma and oligodendroglioma are both gliomas, each taking its name from the central nervous system cells where the cancer starts to grow. These low-grade (slow-growing) gliomas typically affect patients in their 30s and 40s. While these cancers are rare, Servier says diffuse gliomas with IDH mutations are the most common malignant primary brain tumors diagnosed in those age 50 or younger. Surgery is the first-line of treatment, but if these brain cancers develop into high-grade gliomas, treatment options are limited to radiation and chemotherapy — both of which introduce a range of adverse effects.
FDA review of Voranigo was based on the results of a placebo-controlled Phase 3 clinical trial that enrolled 331 patients who already had surgery for Grade 2 astrocytoma or oligodendroglioma. Those cancers were confirmed to have an IDH1 or IDH2 mutation. The trial excluded patients who previously received treatment, including chemotherapy or radiation. Patients who were randomly assigned to receive a placebo were permitted to cross over to the treatment arm after brain imaging confirmed disease progression.
No deaths were reported in either arm. Progressive disease was reported in 28% of patients in the study drug group and in 54% of participants in the placebo arm. The efficacy analysis was supported by the measure of time to the next therapeutic intervention. This time was not reached for the study drug arm and was 17.8 months for the placebo arm. Results from the pivotal study were presented last year during the annual meeting of the American Society of Clinical Oncology and published in the New England Journal of Medicine.
The recommended Voranigo dose for adults is a 40 mg tablet once daily until the disease progresses or toxicity becomes unacceptable. For adolescents, the drug is dosed according to patient weight. The most common adverse reactions reported in the study include fatigue, headache, muscle pain, and diarrhea. Liver toxicity is another risk of the drug and the label and directs clinicians to monitor patients’ liver function.
“As we advance more targeted therapies, identifying mutations and understanding how these mutations impact cancer and its progression are key to helping the right patients find the right treatment, at the right time,” Servier CEO David Lee said in a prepared statement. “We are humbled to lead the field of IDH-mutant inhibition, and we are committed to researching its applicability in glioma and other cancers.”
Voranigo was originally developed by Agios Pharmaceuticals, which had previously developed and commercialized the IDH2 inhibitor Idhifa and the IDH1 inhibitor Tibsovo, both treatments for advanced cases of acute myeloid leukemia. In 2021, as part of a strategy shift to rare disease, Agios sold its oncology business to Servier for $1.8 billion. Under the deal terms, Agios is entitled to a $200 million milestone payment for Voranigo’s approval. The biotech is also due a 15% royalty from Voranigo sales.
Agios has raised cash by striking royalty deals. In 2020, Royalty Pharma acquired Agios’s royalty rights to Idhifa for $255 million. Two years later, Agios sold to Sagard Healthcare Partners the royalty rights to Tibsovo, bringing in $131.8 million. Earlier this year, Royalty Pharma agreed to pay Agios $905 million contingent on Voranigo’s approval. Royalty Pharma projects the brain cancer drug will top $1 billion in peak annual sales, bringing in more than $150 million in annual royalties.
Voranigo is available now. Through a spokesperson, Servier said it is working with Thermo Fisher Scientific on a companion diagnostic to identify patients eligible for the treatment. Servier said the drug will cost $39,881 a month, which amounts to $478,572 a year. The company added that it is working with commercial and government payers, and it expects Voranigo will receive strong coverage based on the strong clinical data that supported its regulatory approval.
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