Barcelona, Spain—Alterations in the fibroblast growth factor receptor (FGFR)2 gene have been identified as driver mutations in cholangiocarcinoma (CCA). Durable objective responses were observed in >33% of patients with locally advanced or metastatic CCA and FGFR2 rearrangements or fusions who received treatment with pemigatinib, a selective oral inhibitor of FGFR1, FGFR2, and FGFR3. Data from the single-arm, open-label phase 2 clinical trial FIGHT-202, which was presented at the ESMO Congress 2019, revealed that investigational pemigatinib induced a response in 35.5% of the 107 patients with FGFR2 fusions or rearrangements (cohort A), with a median duration of response of 7.5 months.
Barcelona, Spain—Ivosidenib (Tibsovo), an oral therapy that targets isocitrate dehydrogenase-1 (IDH1) mutation, significantly improved progression-free survival (PFS) in patients with advanced cholangiocarcinoma (CCA) and an IDH1 mutation, in a phase 3 clinical trial reported lead investigator Ghassan K. Abou-Alfa, MD, Medical Oncologist, Gastrointestinal Oncology Service, Memorial Sloan Kettering Cancer Center, New York City, at the ESMO Congress 2019.
The combination of the investigational drug quizartinib plus azacitidine (Vidaza) or low-dose cytarabine has substantial activity in patients with myeloid leukemias and FLT3 mutations.
Treatment with ivosidenib, an IDH1 inhibitor, resulted in an objective response rate of 41.6% in a phase 1 dose-escalation and expansion clinical trial of patients with relapsed or refractory acute myeloid leukemia and IDH1 mutation, according to data presented at ASH 2017.
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