FDA Pipeline: Fast Track/Orphan Drug Designation for Cholangiocarcinoma Treatment, Breakthrough Device Designation for Bladder Cancer Test

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This week, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to infigratinib for patients with advanced or metastatic cholangiocarcinoma with a certain genetic mutation, and Orphan Drug designation to the treatment for patients with cholangiocarcinoma. The FDA also granted Breakthrough Device designation to a test for the detection of bladder cancer.

Fast Track, Orphan Drug Designation for Infigratinib

The FDA granted Fast Track designation to infigratinib for adults with first-line advanced or metastatic cholangiocarcinoma with FGFR2 (fibroblast growth factor receptor 2) gene fusions or translocations and Orphan Drug designation to infigratinib for the treatment of cholangiocarcinoma. Enrollment is ongoing, and patient dosing has started in the PROOF trial, a phase III clinical trial evaluating oral infigratinib in adults for the first-line treatment of advanced cholangiocarcinoma with FGFR2 gene fusions or translocations.

FGFR2 genetic aberrations are present in approximately 15% to 20% of people who have this disease. Currently, treatment options are limited, and the 5-year survival rate is 9%.

The PROOF trial will enroll approximately 384 patients with first-line cholangiocarcinoma with FGFR2 fusions or translocations as determined by molecular profiling. The primary endpoint is progression-free survival compared to standard-of-care chemotherapy (gemcitabine and cisplatin). Patients will be randomly assigned 2:1 to infigratinib vs standard of care.

“Importantly, in this trial, patients who are assigned to receive standard of care will be allowed to cross over and receive infigratinib if they do not respond to chemotherapy,” said Stacie Lindsey, President of the Cholangiocarcinoma Foundation. “Having a crossover option is very significant to patients.”

For additional information on the PROOF trial, including eligibility, patients should ask their physician, visit, or e-mail

Breakthrough Device Designation for URO17, Innovative Urine Test for Bladder Cancer

The FDA granted Breakthrough Device designation for the URO17 bladder cancer recurrence test. This new test exploits a novel cancer marker—keratin 17—that could improve the accuracy and sensitivity of noninvasive bladder cancer testing through urinalysis.

An initial study by Babu et al published in Modern Pathology showed the URO17 test exhibited 100% sensitivity and 96% specificity in detecting recurrent bladder cancer from urine samples.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.