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Incyte's Pemazyre, Foundation Medicine CDx Get FDA Approval for FGFR2-Mutated Bile Duct Cancer

NEW YORK – Incyte received US Food and Drug Administration's approval for pemigatinib (Pemazyre) on Friday for previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements.

Cholangiocarcinoma is a rare cancer of the bile ducts that occurs in approximately 8,000 patients in the US annually. FGFR2 fusions and rearrangements occur in between 9 percent and 14 percent of cholangiocarcinoma patients.

The drug was approved alongside Roche business Foundation Medicine's FoundationOne CDx as a companion test for identifying this subset of patients. With the latest approval, the next-generation sequencing panel has been approved as a CDx alongside 20 cancer drugs. 

The FDA granted orphan therapy and breakthrough therapy designation, as well as priority review to pemigatinib. The agency approved the drug based on results of the Phase II, single-arm FIGHT-202 trial, which involved three cohorts: one involving patients with FGFR2 fusions or rearrangements; a second including patients with other types of FGF or FGFR genetic alterations; and a third with patients with no FGF or FGFR genetic alterations. 

The study showed that 36 percent of 107 previously treated patients with FGFR2 fusions or rearrangements saw their tumors shrink. Nearly 3 percent of patients saw their tumors completely disappear, while 33 percent saw partial shrinkage. Of the 38 patients who responded, 24 did so for six months and seven patients responded for a year or longer.

"With [pemigatinib], we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumors harboring FGFR2 gene fusions and other rearrangements to be favorable, particularly when we considered that these patients have no other good options following first-line treatment with chemotherapy," Richard Pazdur, director of the FDA's Oncology Center of Excellence, said in a statement announcing the approval.

The agency also noted that a condition of pemigatinib's accelerated approval is that Incyte will conduct a randomized-controlled trial in the post-market setting and show that the drug improves progression-free and overall survival over a comparator. Continued approval of pemigatinib may be contingent on the results of this study.

In the trial, 20 percent of patients commonly experienced electrolyte disorders, alopecia, diarrhea, nail toxicity, and fatigue. Pemigatinib treatment also carries with it the risk of ocular toxicity, the FDA said.

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