FDA Approves Pemigatinib as First-to-Treatment Targeted Therapy for Adult Patients with Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma
April 17, 2020, Wilmington, Delaware, BUSINESS WIRE.
Incyte Corporation announced that the U.S. Food and Drug Administration (FDA) has approved pemigatinib, a kinase inhibitor, for the treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma who have been confirmed by FDA-approved testing to have fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Pemigatinib is the first and only drug approved by the FDA for this indication, granted under the accelerated approval pathway based on overall response rate (ORR) and duration of response (DOR). Further approval may depend on the validation and description of clinical benefit in confirmatory trials.
Hervé Hoppenonot, CEO of Incyte, stated, “Our series of studies on FGFR2 fusions or rearrangements in cholangiocarcinoma and the development of pemigatinib as a first-targeted treatment option perfectly exemplify how we translate scientific findings into something that can truly have a positive impact on patients’ lives. With the successful approval of pemigatinib, we bring new hope to patients for better treatment options, immediately adding it to the lineup of new treatment choices in clinical practice.”
The FDA approved pemigatinib based on preliminary data from the multicenter, open-label, single-arm FIGHT-202 clinical trial, which studied the efficacy of pemigatinib in adult patients with cholangiocarcinoma. Preliminary results from the clinical trial showed a 36% overall response rate and a median duration of response of 9.1 months in patients with FGFR2 fusions or rearrangements as monotherapy.
Precautions for Pemigatinib: Eye problems (such as dry or inflamed eyes, corneal inflammation, increased tearing, and retinal disturbances); elevated blood phosphate levels; and the risk of fetal harm or miscarriage in pregnant women.
The FDA has granted pemigatinib Breakthrough Therapy Designation for the treatment of previously treated patients with advanced/metastatic or unresectable FGFR2 translocation cholangiocarcinoma. The FDA has also granted pemigatinib Orphan Drug Designation for the treatment of cholangiocarcinoma, and its New Drug Application (NDA) is under review through the FDA's Priority Review process.
Experts at Ghassan Abou-Alfa Sloan Kettering Cancer Center stated, "Although cholangiocarcinoma is considered a rare malignancy, its incidence has been steadily rising over the past three decades, now accounting for more than 20% of pancreatic cancers. New targeted therapy options are encouraging for patients with limited choices after first-line chemotherapy or surgery, although recurrence rates remain high."
Chlorochocarcinoma Overview: Cholangiocarcinoma is a rare cancer that forms in the bile ducts. Based on its anatomical origin, it is divided into two types: intrahepatic cholangiocarcinoma (iCCA), which occurs in the bile ducts within the liver, and extrahepatic cholangiocarcinoma, which occurs in the bile ducts outside the liver. Patients with cholangiocarcinoma are usually diagnosed at an advanced stage or with a poor prognosis. In North America and Europe, the incidence of cholangiocarcinoma varies considerably by region, ranging from approximately 0.3 to 3.4 cases per 100,000 people. FGFR2 fusions or rearrangements are almost exclusively seen in iCCA, accounting for approximately 10%-16% of patients. FGFRs play an important role in tumor cell proliferation and survival, migration, and angiogenesis (neovascularization). Activation, fusion, rearrangement, translocation, and gene amplification of FGFRs are closely associated with the development of various cancers.
"The approval of pemigatinib provides patients with an exciting new treatment option, bringing hope to those who often face a difficult diagnostic journey and poor prognosis," said Stacie Lindsey, President of the Cholangiocarcinoma Foundation.
Incyte is committed to supporting patients and removing barriers to accessing medicines. Eligible patients in the U.S. who are prescribed pemigatinib can receive support through the Incyte CARES (Connecting Access, Reimbursement, Education, and Support) program, a comprehensive program that provides eligible patients with ongoing education and resources, including financial assistance. For more information, please visit pemigatinib.com.
The FDA is also expected to approve FoundationOne® CDx as a companion diagnostic for pemigatinib. With its in-depth whole-genome analysis, FoundationOne CDx provides a comprehensive genomic interpretation for companion diagnostics of all solid tumors. If approved, this companion diagnostic will help identify patients with FGFR2 fusions or rearrangements who may be suitable for pemigatinib treatment.
