NEW YORK – Cambridge, Massachusetts-based Agios Pharmaceuticals on Friday announced that it had withdrawn its marketing application with the European Medicines Agency for ivosidenib (Tibsovo) for the treatment of patients with IDH1-mutated relapsed or refractory acute myeloid leukemia.
The withdrawn application comes in the wake of negative feedback from the EMA's Committee for Medicinal Products for Human Use (CHMP). In CHMP's view, there is insufficient evidence demonstrating ivosidenib's benefit in this specific indication. The application had hinged on data from a single-arm, Phase I clinical trial involving 174 patients with IDH1-mutated, relapsed or refractory AML. After a median follow-up time of 8.3 months, roughly 33 percent of patients achieved either complete remission or complete remission with partial hematological recovery.
In contrast to the EMA, the US Food and Drug Administration approved ivosidenib for this indication based on these data in 2018.
"We are disappointed not to be able to bring Tibsovo to AML patients in the EU based on our current data in the relapsed/refractory setting, given the tremendous need for new targeted treatment options for this devastating disease," Agios Chief Medical Officer Chris Bowden said in a statement.
Bowden added that contingent on the outcomes of several ongoing Phase III randomized trials, Agios may still seek marketing authorization for ivosidenib in combination with other agents in AML. In one Phase III trial, Agios is evaluating ivosidenib plus chemotherapy versus chemo for patients with newly diagnosed, IDH1-mutated AML, and in another Phase III trial it is comparing ivosidenib against enasidenib (Bristol Myers Squibb/Agios' Idhifa) in combination with standard induction and consolidation chemotherapy for patients with newly diagnosed, IDH1-mutated AML.