FDA Grants Priority Review to Pemigatinib for Cholangiocarcinoma Treatment

The FDA has granted a priority review to a new drug application (NDA) for pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma — a cancer that originates in the bile ducts – with FGFR2 fusions or rearrangements, according to Incyte, the agent’s manufacturer.

An NDA is granted when a pharmaceutical company asks the FDA to approve a drug based on successful clinical trial results, and a priority review will expedite the timeline in which the agency will review — and potentially approve – the agent.

“There is a significant need for new therapies for patients with cholangiocarcinoma, who have limited treatment options beyond first-line chemotherapy and often face a poor prognosis,” Peter Langmuir, M.D., group vice president, targeted therapeutics, Incyte, said in a press release.

“We are very pleased that the FDA has accepted our NDA for priority review which we believe represents an important step toward providing the first treatment option for patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. We intend to work closely with the FDA to bring this innovative targeted therapy to patients suffering from this devastating disease as soon as possible.”

The NDA was based on data from the phase 2, open-label, multicenter FIGHT-202 study (NCT02924376), designed to evaluate the efficacy and safety of pemigatinib in patients with previously treated, locally advanced or metastatic cholangiocarcinoma.

Patients were enrolled into one of three cohorts:

• cohort A (patients with FGFR2 fusions or rearrangements)
• cohort B (patients with other FGF/FGFR genetic alterations)
• cohort C (patients with no FGF/FGFR genetic alterations).

All patients received 13.5 mg of pemigatinib orally once daily on a 21-day cycle that was two weeks on followed by one week off, until radiological disease progression or unacceptable side effects.

The primary endpoint overall response rate (ORR) in cohort A, assessed by independent review per RECIST v1.1; while secondary endpoints included ORR in cohorts B, A plus B, and C; progression free survival (the length of time a patient goes without disease progression), overall survival (total time between treatment and death), duration of response (DOR) (the time a patient responds to a treatment before progression/death), disease control rate, and safety in all cohorts.

Study results were presented at the European Society for Medical Oncology (ESMO) 2019 Congress.

Patients harboring FGFR2 fusions or rearrangements (cohort A) showed an ORR of 36% and DOR of 7.5 months with pemigatinib alone after an average follow-up of 15 months.

Side effects were manageable and consistent with pemigatinib, according to the release.

The Prescription Drug User Fee Act (PDUFA) target action date — which is when the FDA must make its decision on whether or not to approve the drug – is May 30, 2020.