April 17, 2020

The U.S. FDA has approved Pemazyre™ (pemigatinib), manufactured by Incyte, for use in adults who have 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. It is the first and only drug to receive FDA approval for this indication.

Cholangiocarcinoma is a rare cancer that begins in a series of thin tubes that go from the liver to the small intestine called bile ducts. It is often not diagnosed until it has reached a late or advanced stage and the patient’s prognosis is poor. FGFR2 fusions or rearrangements help to drive the survival, growth, and proliferation of the cancer’s tumors. Premazyre is designed to specifically target cancer cells that have these fusions and rearrangements.

In a clinical study, Pemazyre given as a monotherapy delivered an overall response rate of 36% and median duration of response of 9.1 months. Of the patients who responded to treatment, 63% had a response that lasted for at least six months, while 18% had a response lasting 12 months or longer.

Recommended dosing is one 13.5mg tablet taken by mouth once daily for 14 consecutive days, with or without food. The 14-day period should be followed by seven days off therapy, creating a 21-day cycle. Treatment should continue until disease progression or unacceptable toxicity occurs.

Because the FDA granted accelerated approval to Pemazyre, continued approval may be contingent on verification and description of clinical benefit in confirmatory trials. The product is available at a wholesale acquisition cost (WAC) of $17,000 per 14 tablets.

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