Rare Daily Staff

Incyte said that the U.S. Food and Drug Administration has accepted for Priority Review its new drug application for its experimental drug pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma.

The application seeks approval of pemigatinib for patients with fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements.

The FDA grants Priority Review to medicines that may offer a major advance in treatment where none currently exists. This designation shortens the review period to six months compared to 10 months for Standard Review. The Prescription Drug User Fee Act (PDUFA) target action date is May 30, 2020.

Cholangiocarcinoma is a rare cancer that forms in the bile duct. It is classified based on its origin: intrahepatic cholangiocarcinoma occurs in the bile duct inside the liver and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver. Patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor. FGFR2 fusions or rearrangements occur almost exclusively in intrahepatic cholangiocarcinoma and are found in 10 to 16 percent of those patients.

Fibroblast growth factor receptors play an important role in tumor cell proliferation and survival, migration and the formation of new blood vessels to fuel tumor growth. Activating fusions, rearrangements, translocations, and gene amplifications in FGFRs are closely correlated with the development of various cancers. Pemigatinib is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3, which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.

The new drug application submission is based on data from the FIGHT-202 study evaluating pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma. Study results demonstrated that in patients harboring FGFR2 fusions or rearrangements, pemigatinib monotherapy resulted in an overall response rate of 36 percent, and median duration of response of 7.5 months with a median follow-up of 15 months. Adverse events were manageable and consistent with the mechanism of action of pemigatinib.

“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,” said Peter Langmuir, group vice president of targeted therapeutics at Incyte. “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 U.S. Food and Drug Administration has previously granted pemigatinib Breakthrough Therapy designation for the treatment of previously treated, advanced/metastatic or unresectable FGFR2 translocated cholangiocarcinoma. The agency also granted pemigatinib Orphan Drug designation for the treatment of cholangiocarcinoma.

Photo: Peter Langmuir, group vice president of targeted therapeutics at Incyte

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