FDA grants accelerated approval to pemigatinib for cholangiocarcinoma with an FGFR2 rearrangement or fusion

On April 17, 2020, the Food and Drug Administration granted accelerated approval to pemigatinib (PEMAZYRE, Incyte Corporation) for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

The FDA also approved the FoundationOne® CDX (Foundation Medicine, Inc.) as a companion diagnostic for patient selection.

Efficacy was investigated in FIGHT-202 (NCT02924376), a multicenter open-label single-arm trial, in 107 patients with locally advanced unresectable or metastatic cholangiocarcinoma whose disease had progressed on or after at least one prior therapy and had an FGFR2 gene fusion or rearrangement (clinical trial assay performed at a central laboratory). Patients received pemigatinib, 13.5 mg orally, once daily for 14 consecutive days, followed by 7 days off therapy.

The major efficacy outcome measures were overall response rate (ORR) and duration of response (DOR) determined by an independent review committee using RECIST 1.1. Among the 107 patients, the ORR was 36% (95% CI: 27%, 45%), including 3 complete responses. The median DOR was 9.1 months with responses lasting ≥ 6 months in 24 of the 38 (63%) responding patients and ≥ 12 months in 7 (18%) patients.

The most common adverse reactions to pemigatinib (incidence ≥ 20%) were hyperphosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, arthralgia, abdominal pain, hypophosphatemia, back pain, and dry skin. Ocular toxicity and hyperphosphatemia are important risks of pemigatinib.

The recommended pemigatinib dose is 13.5 mg orally once daily for 14 consecutive days followed by 7 days off therapy in 21-day cycles.

View full prescribing information for PEMAZYRE.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

This application was granted priority review, breakthrough therapy and orphan drug designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

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