US-based biopharmaceutical company Incyte has announced that the US Food and Drug Administration (FDA) accepted for priority review its New Drug Application (NDA) for pemigatinib as a treatment for patients with cholangiocarcinoma.
Cholangiocarcinoma is cancer that forms in the bile duct, with patients often diagnosed at a late or advanced stage.
Pemigatinib is a selective fibroblast growth factor receptor (FGFR) inhibitor with the potential to treat patients with previously managed, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements.
Incyte has submitted the NDA based on data from the FIGHT-202 study that evaluated pemigatinib as a treatment for cholangiocarcinoma patients.
For the FIGHT-202 Phase II study, patients enrolled into Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF / FGFR genetic alterations) or Cohort C (no FGF / FGFR genetic alterations).
The FIGHT-202 Phase II, open-label, multicentre study evaluates the safety and efficacy of pemigatinib in adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF / FGFR status.
Patients were administered 13.5mg pemigatinib orally once daily (QD) on a 21-day cycle until radiological disease progression or unacceptable toxicity.
The primary endpoint of FIGHT-202 is the overall response rate (ORR) in Cohort A. Secondary endpoints include ORR in Cohorts B, A plus B, and C, as well as progression-free survival (PFS), overall survival (OS), duration of response (DOR), disease control rate (DCR) and safety across cohorts.
FGFRs are said to play an essential role in tumour cell proliferation and survival, migration and angiogenesis.
Pemigatinib is a potent inhibitor of FGFR isoforms 1, 2 and 3, which has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations
Results showed that in patients with FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy yielded an overall response rate (ORR) of 36% (primary endpoint). Furthermore, pemigatinib also resulted in a median duration of response (DOR) of 7.5 months (secondary endpoint) with a median follow-up of 15 months.
The company said that adverse events were manageable and consistent with pemigatinib’s action.
The Fibroblast Growth factor receptor in oncology and Haematology Trials (FIGHT) clinical trial programme includes ongoing Phase II and III studies investigating the safety and efficacy of pemigatinib therapy across FGFR-driven malignancies.
The FDA also granted pemigatinib Orphan Drug designation for the treatment of cholangiocarcinoma.