FDA grants priority review to ivosidenib for leukemia subtype
The FDA granted priority review to ivosidenib for treatment of relapsed or refractory acute myeloid leukemia with IDH1 mutation, according to the agent’s manufacturer.
Ivosidenib (AG-120, Agios) is an oral targeted inhibitor of mutant IDH1.
The FDA based the priority review on results from the AG120-C-001 trial, a phase 1 dose-escalation expansion study of ivosidenib that included 258 patients with IDH1 mutation.
Results of this study were presented in December at ASH Annual Meeting and Exposition.
As HemOnc Today previously reported, the overall response rate was 41.6% (95 CI%, 32.9-50.8) and median time to first response was 1.9 months (95% CI, 0.8-4.7). Median duration of response was 6.5 months (95% CI, 4.6-9.3).
Median OS was 8.8 months (95% CI, 6.7-10.2) among all patients, with best response observed in relapsed/refractory AML (n = 125).
“After decades of little change, treatment of AML has begun to shift dramatically as result of new therapies, and IDH inhibitors will play an important role in how we treat this terrible disease,” David Schenkein, MD, CEO of Agios, said in a company-issued press release. “Today marks an important milestone in our efforts to rapidly advance what could be the first targeted treatment for [relapsed/refractory] AML patients with an IDH1 mutation. We appreciate the FDA’s collaboration during the application process, and we look forward to continuing our productive dialogue.”
Additionally, Abbott submitted a premarket approval application for an IDH1 assay (m2000 RealTime System, Abbott) that will act as a companion diagnostic for ivosidenib. Abbott and Agios previously entered into an exclusive agreement to develop this assay to use with the IDH1 inhibitor.
The FDA is scheduled to make a decision on ivosidenib’s status for this indication by August.