The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Nuvation Bio’s safusidenib for the treatment of IDH1-mutant glioma. The news was shared in a recent news release by Nuvation Bio.
Safusidenib is an investigational, oral, brain-penetrant therapy designed to selectively inhibit mutant IDH1. The Fast Track designation is intended to facilitate the development and expedite the review of therapies for serious conditions with unmet medical needs. It does not constitute FDA approval of safusidenib.
The designation was supported by favorable data from the safusidenib clinical development program, including updated long-term results from the Phase 2 J201 study in 27 patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant glioma in Japan.
At a median follow-up of 38.8 months, the centrally assessed confirmed objective response rate was 51.9%. Median progression-free survival had not been reached, while the 36-month progression-free survival rate was 79.1%. Only one patient who had previously responded subsequently experienced disease progression, and no new safety signals were identified with longer follow-up.
About Safusidenib
Safusidenib is an investigational, oral, brain-penetrant, selective inhibitor of mutant IDH1. It is being developed for patients with IDH1-mutant glioma, including settings where targeted treatment options remain limited.
In Phase 1 and Phase 2 studies, the drug has demonstrated clinical activity, including delayed disease progression and durable responses across different tumor grades and risk groups. Safusidenib is currently being evaluated across several clinical studies, including the pivotal Phase 3 SIGMA trial.
In August 2026, the FDA granted safusidenib Fast Track Designation for IDH1-mutant glioma.
About the Phase 3 SIGMA Trial
SIGMA (NCT05303519) is a pivotal Phase 3, multicenter study evaluating the efficacy and safety of safusidenib in patients with IDH1-mutant glioma.
The pivotal portion of the study is evaluating safusidenib versus placebo as maintenance therapy following standard-of-care treatment in patients with IDH1-mutant astrocytoma with high-risk features. Approximately 300 patients are expected to be enrolled in this portion of the study.
The study also includes a separate exploratory, non-pivotal cohort evaluating safusidenib in approximately 40 patients with grade 3 IDH1-mutant oligodendroglioma who have undergone surgery but have not yet received chemotherapy or radiotherapy.
Enrollment in the Phase 3 SIGMA study is currently underway.
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