According to a press release from Servier Pharmaceuticals, the U.S. Food and Drug Administration (FDA) has accepted and granted priority review to the supplemental New Drug Application (sNDA) for TIBSOVO (ivosidenib) for the treatment of patients with relapsed or refractory (R/R) myelodysplastic syndrome (MDS) due to isocitrate dehydrogenase 1 (IDH1) mutations.
TIBSOVO is currently approved in the United States as a monotherapy for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) due to isocitrate dehydrogenase 1 (IDH1) mutations, and as a monotherapy or in combination with azacitidine for newly diagnosed adults aged ≥75 years with isocitrate dehydrogenase 1 (IDH1)-mutant AML or adults with comorbidities excluding those who have received intensive induction chemotherapy.
In addition, TIBSOVO was recently approved by the European Commission as a targeted therapy for two indications: in combination with azacitidine for the treatment of newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation in adult patients who are ineligible for standard induction chemotherapy; and as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who have previously received at least one prior systemic therapy.
TIBSOVO has also been approved in the United States and Australia for patients with previously treated IDH1-mutant cholangiocarcinoma. In China, TIBSOVO is approved for the treatment of adult patients with relapsed or refractory AML who are susceptible to IDH1 mutations.
The supplemental New Drug Application for the MDS indication is supported by data from a Phase 1 open-label study (ClinicalTrials.gov identifier: NCT02074839) that included 18 patients with relapsed or refractory MDS with IDH1 mutations.
Results showed that TIBSOVO achieved an objective response rate of 83.3%, with 38.9% of patients achieving complete remission (CR). The median time to complete remission was 1.87 months (range 1.0–5.6). At the time of analysis, the median duration of non-CR was 1.9–80.8 months, and the median overall survival was 35.7 months (range 3.7–88.7 months).
Of the nine patients who were dependent on erythrocyte and/or platelet transfusions at baseline, six (66.7%) became transfusion-independent within a period of ≥56 days post-baseline. No new safety signals were reported in the study.
If approved, TIBSOVO will be the first and only approved targeted therapy for patients with MDS susceptible to IDH1 mutations.
