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On March 30, the U.S. Food and Drug Administration (FDA) has been subject to priority review of a Futibatinib New Drug Application (NDA) for the treatment of a previously treated patient with locally advanced or metastatic cholangiocarcinoma with an FGFR2 gene rearrangement, including gene fusion.
Futibatinib is a research,orally potent, potent, selective, and irreversible small molecule inhibitor of FGFR 1, 2, 3, and 4. The NDA submission is supported by data from the ongoing critical, open-label Phase 2b FOENIX-CCA2 trial (clinical trial). gov identifier: NCT02052778) evaluated futibatinib in 103 adults with locally advanced or metastatic cholangiocarcinoma who had fGFR2 gene rearrangements, including gene fusion that had undergone one or more previous systemic therapies. Patients take futibatinib 20 mg orally once a day until the disease progresses or an unacceptable toxicity develops.
Results showed an objective response rate of 41.7% (primary endpoint) for treatment with futibatinib as assessed by an independent central review. The median duration of response (secondary endpoint) was 9.7 months, with 72% of responses lasting at least 6 months.
As for safety, common treatment-related adverse events were hyperphosphatemia (85%), hair loss (33%), and dry mouth (30%). More than 1 patient reported a serious adverse reaction of migraine (1.9%).
A target date of September 30, 2022 for the Prescription Drug User Fee Act has been set for this application. "Given the lack of recognized standard chemotherapy after first-line treatment failures, futibatinib may represent an important opportunity for targeted therapies for this subset of CCA patients, which is driving our pursuit of this research compound," Volker Wacheck said. ”
According to trial data, in 2021, the FDA granted futibatinib breakthrough therapy designation for the treatment of patients with locally advanced or metastatic cholangiocarcinoma who had previously been treated.