Europe has taken five years longer than the United States to approve ivosidenib, sold as Tibsovo, for acute myeloid leukemia (AML). The drug first won U.S. approval in July 2018. Its arrival in the European market, combined with chemotherapy, gives some patients with this aggressive blood cancer a new option.
Ivosidenib blocks the activity of the IDH1 gene. According to Medindia, which covered the approval on May 22, 2023, that has translated into higher overall survival and complete remission rates. Until now, patients with FLT3 mutations were the only group in Europe with a targeted treatment available.
Why Europe lagged
The U.S. approval in 2018 covered AML in both the first-line and relapsed or refractory settings, and relapsed or refractory cholangiocarcinoma as well. China followed in 2022, when CStone Pharma received NMPA approval to bring the drug to that market.
Europe’s wait traces back to 2020. That year the manufacturer voluntarily withdrew its application to use ivosidenib alone against AML, after the European authority judged data from the Phase III Study AG120-C-001 insufficient for market authorization.
The same hurdle may slow two other drugs, Idhifa and Rezlidhia. Their clinical trials used complete remission rates as the primary efficacy endpoint, and that choice could stand in the way of European approval.
A thinner field in bile duct cancer
Tibsovo also faces less competition in cholangiocarcinoma. In Europe, 18 drug candidates are in clinical development for that indication, from Phase I to Phase III. AML has 33.
