New York-based Nuvation Bio Inc. (NYSE: NUVB) announced that the US FDA has granted Fast Track Designation to safusidenib, an oral, brain-penetrant, selective inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), for the treatment of IDH1-mutant glioma.
The designation was granted on the basis of Phase II data from the J201 study, a single-arm trial enrolling 27 patients in Japan with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant glioma. At a median follow-up of 38.8 months, safusidenib produced a centrally assessed confirmed objective response rate (cORR) of 51.9% per RANO-LGG criteria. Median progression-free survival (PFS) was not reached, with a 36-month PFS rate of 79.1%. Only one patient who had previously responded experienced subsequent disease progression, and no new safety signals were identified with extended follow-up. These data, published in Neuro-Oncology, build on an earlier interim analysis at a March 2023 cutoff that reported a 44.4% confirmed ORR and an 87.9% event-free probability at 24 months.
Safusidenib enters a therapeutic class already validated by Servier's Voranigo (vorasidenib), a brain-penetrant dual IDH1/IDH2 inhibitor approved by the FDA in 2024 for grade 2 IDH-mutant glioma following surgery. Safusidenib selectively targets mutant IDH1, but whether that pharmacological distinction translates into a clinical advantage remains unclear. Its pivotal SIGMA program is also pursuing a different setting, evaluating maintenance treatment after standard therapy in higher-risk IDH1-mutant astrocytoma.