Back to press releases

Foundation Medicine Receives FDA Approval for FoundationOne®CDx as the Companion Diagnostic for Pemazyre™ (pemigatinib), the First FDA-Approved Targeted Therapy for Adults with Previously Treated Locally Advanced or Metastatic Cholangiocarcinoma

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Foundation Medicine, Inc. today announced that the U.S. Food and Drug Administration (FDA) approved FoundationOne®CDx as the registrational companion diagnostic for Incyte’s Pemazyre™ (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor approved for adults with 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. FoundationOne CDx is the first and only FDA-approved companion diagnostic test for this indication.

Cholangiocarcinoma is a rare cancer, diagnosed in approximately 8,000 patients annually, according to the American Cancer Society. 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. FGFR2 fusions or select rearrangements occur in 10-16 percent of iCCA patients.1, 2, 3 Because this cancer is difficult to detect, patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor and treatment options are limited.4 The approval of this novel therapy and companion diagnostic means healthcare professionals will be able to use Foundation Medicine’s comprehensive genomic profiling (CGP) assay, FoundationOne CDx, to identify patients with FGFR2 fusions and select rearrangements who may benefit from treatment with Pemazyre in accordance with the approved therapeutic product labeling.

“The approval of this therapy and companion diagnostic is an important step forward in advancing care for patients with cholangiocarcinoma for whom there are limited treatment options,” said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. “The breadth and depth of our sequencing allows for identification of patients with this rare fusion who may benefit from Pemazyre, underscoring the importance of comprehensive genomic profiling in cholangiocarcinoma. We continue to work toward broadening patient access to biomarker-driven therapies, illustrated by this recent milestone. We are proud to add another important FDA-approved companion diagnostic to FoundationOne CDx.”

FoundationOne CDx is the first FDA-approved broad companion diagnostic that is clinically and analytically validated for solid tumors. It is currently approved as the companion diagnostic test for 20 unique therapies across multiple cancer types.

“Patients with rare cancers like cholangiocarcinoma often face additional challenges when it comes to available research and treatment options for their condition,” said Jim Palma, Executive Director at Target Cancer Foundation. “The approval of this therapy and companion diagnostic is a turning point for these patients and the rare cancer community as a whole. Not only could it be lifechanging for cholangiocarcinoma patients with FGFR2 fusions and select rearrangements, but it reinforces the critical need for comprehensive genomic profiling and the power of collaboration among scientists, clinicians, patients and advocacy organizations.”

Incyte announced earlier today the U.S. FDA approval of Pemazyre as the first selective FGFR inhibitor for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test. Pemazyre 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. More information about Pemazyre can be found at

About FoundationOne CDx

FoundationOne CDx is a next-generation sequencing based in vitro diagnostic device for detection of substitutions, insertion and deletion alterations (indels), and copy number alterations (CNAs) in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumor tissue specimens. FoundationOne CDx is intended as a companion diagnostic to identify patients who may benefit from treatment with certain targeted therapies in accordance with their approved therapeutic product labeling. Additionally, FoundationOne CDx is intended to provide tumor mutation profiling to be used by qualified health care professionals in accordance with professional guidelines in oncology for patients with solid malignant neoplasms. For a full list of targeted therapies for which FoundationOne CDx is indicated as a companion diagnostic, please visit

About FGFR

Fibroblast growth factor receptors (FGFRs) play an important role in tumor cell proliferation and survival, migration and angiogenesis (the formation of new blood vessels). Activating fusions, rearrangements, translocations and gene amplifications in FGFRs are closely correlated with the development of various cancers.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patient’s cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicine’s molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine®and FoundationOne® are registered trademarks of Foundation Medicine, Inc.

Pemazyre™ is a trademark of Incyte.

Source: Foundation Medicine

1 Graham RP, et al. Hum Pathol. 2014;45:1630‒1638.
2 Farshidfar F, et al. Cell Rep. 2017;18(11):2780–2794.
3 Ross JS et al. The Oncologist. 2014;19:235–242.
4 Uhlig J, et al. Ann Surg Oncol. 2019;26:1993–2000.

Foundation Medicine Contact:
Lee-Ann Murphy, 617-245-3077

Source: Foundation Medicine

April 18, 2020