FDA授予了futibatinib治疗胆管癌的突破性治疗资格认定

The FOENIX-CCA2 trial actually enrolled 386 patients and consisted of a single-arm trial. In the single-arm trial, patients received futibatinib orally for 21 days (one cycle). The primary outcome of the Phase 2 trial was overall response rate (ORR). Secondary outcomes include duration of response (DOR), disease control rate (DCR), progression-free survival (PFS), patient-reported outcome (PRO), and overall survival (OS).

To be included in the trial, patients must be at least 18 years old and must have failed at least one dose of systemic gemcitabine and platinum-based chemotherapy. In addition, patients must have good organ function. Patients with non-tumor-related signs of altered calcium and phosphorus homeostasis or significant ectopic mineralization/calcification were not eligible for the trial.

Of the 103 patients enrolled in the Phase 2 trial, 67 patients were included in the interim analysis of the FOENIX-CCA2 trial. Among these patients, 82.1% had tumors with FGFR2 mutations. Of the patients evaluated, 44% had received 1 treatment, 28.4% had received 2 treatments, and 26.9% had received 3 treatments. The ORR was 34.3% and the DCR was 76.1%. The median time to response was 1.6 months, and the median DOR was 6.2 months.

Overall, the safety profile was consistent with the drug class. All patients experienced treatment-related adverse events (TRAEs), with 57% of patients experiencing grade 3 adverse events. Serious adverse reactions occurred in 10% of patients, but all but one adverse event occurred only once. Hyperphosphatemia was the most common adverse event (AE) below grade 3, but all cases resolved with medical treatment, and no patient discontinued treatment due to hyperphosphatemia. The incidence rate of grade 3 or above adverse events was 73.1%. Sixty-five percent of patients required dose delays and 53.7% required dose reductions. A total of 6% of patients discontinued treatment due to adverse events.

"Patients with locally advanced and metastatic cholangiocarcinoma currently have poor prognoses, particularly because there are no standard treatments after failure of first-line chemotherapy," said Martin J. Birkhofer, MD, senior vice president and chief medical officer of Taiho Oncology. "We are pleased that the FDA has recognized the potential benefit of futibatinib in patients with previously treated CCA. We are committed to the global market for futibatinib in patients with cholangiocarcinoma and look forward to continued dialogue with the FDA and other health authorities."

futibatinib was originally granted orphan drug designation by the FDA in May 2018. Futibatinib has not been approved by any authority for the treatment of patients other than for investigational purposes.

References:

https://www.targetedonc.com/view/fda-grants-breakthrough-therapy-designation-for-cholangiocarcinoma-treatment-futibatinib