CHOLANGIOCARCINOMA The FDA have granted priority approval review of pemigatinib for patients harbouring FGFR2 fusions.

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Dec. 9, 2019

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CHOLANGIOCARCINOMA

The FDA have granted priority approval review of pemigatinib for patients harbouring FGFR2 fusions, based on interim data from the phase II FIGHT-202 study showing ORR 36%.

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Incyte Announces Acceptance and Priority Review of NDA for Pemigatinib as a Treatment for Patients with Cholangiocarcinoma 27 November, 2019 PDF Version WILMINGTON, Del.--(BUSINESS WIRE)--Nov. 27, 2019-- Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its New Drug Application (NDA) for pemigatinib, a selective fibroblast growth factor receptor (FGFR) inhibitor, as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements. The NDA 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, recently presented at the European Society for Medical Oncology (ESMO) 2019 Congress, demonstrated that in patients harboring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate (ORR) of 36 percent (primary endpoint), and median duration of response (DOR) of 7.5 months (secondary endpoint) 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, M.D., Group Vice President, Targeted Therapeutics, Incyte. “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 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 eight months compared to 12 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 (iCCA) 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.1,2 The incidence of cholangiocarcinoma varies regionally and ranges between 0.3 – 3.4 per 100,000 in North America and Europe.1 FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16 percent of patients.3-5 About FIGHT-202 The FIGHT-202 Phase 2, open-label, multicenter study (NCT02924376) is evaluating the safety and efficacy of pemigatinib – a selective fibroblast growth factor receptor (FGFR) inhibitor – in adult (age ≥ 18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF/FGFR status. Patients were enrolled into one of three cohorts – Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genetic alterations) or Cohort C (no FGF/FGFR genetic alterations). All patients received 13.5 mg pemigatinib orally once daily (QD) on a 21-day cycle (two weeks on/one week off) until radiological disease progression or unacceptable toxicity. The primary endpoint of FIGHT-202 is overall response rate (ORR) in Cohort A, assessed by independent review per RECIST v1.1. Secondary endpoints include ORR in Cohorts B, A plus B, and C; progression free survival (PFS), overall survival (OS), duration of response (DOR), disease control rate (DCR) and safety in all cohorts.