FDA Grants Orphan Drug Designation to Cholangiocarcinoma Therapy

Rebekah Harrison

The FDA granted orphan drug designation to ArQule’s, ARQ 087 for the treatment of cholangiocarcinoma.
ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family. FGFR translocations have been identified as a potential target for tyrosine kinase inhibitor therapies.

What is Cholangiocarcinoma?

Cholangiocarcinoma is a cancerous growth in one of the ducts that caries bile to the liver. Symptoms include fever, chills, itching, loss of appetitde, abdomen pain and jaundice.
Incidence in most Western countries ranges from 2 to 6 cases per 100,000 people per year.
ArQule is currently conducting a biomarker-driven phase 2 trial for ARQ 087 in the US and Italy in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR fusions. The commencement of the phase 2 trial followed the observation of 2 confirmed partial responses in iCCA with FGFR fusions in the phase 1a portion of the trial. A minor response was also observed in a third patient also harboring an FGFR fusion.
The phase 2 trial is designed to enroll up to 20 patients and potentially provide a clinical signal to be further explored in an expedited clinical strategy in this rare indication.
“This year we have already received orphan drug designation for ARQ 092 in Proteus syndrome. We are now pleased to have received our second orphan drug designation from the FDA for ARQ 087 in CCA. This is part of our continued pursuit of precision medicine,” said Brian Schwartz, MD, head of Research and Development and chief medical officer at ArQule. “CCA is a rare cancer that lacks approved therapeutic options to specifically target FGFR2 fusions. We are very encouraged by the clinical efficacy so far observed in the phase 1 portion of the trial which validates the pre-clinical data recently presented at the AACR-NCI-EORTC conference that demonstrated efficacy across a number of FGFR2-driven tumors in xenograft models.”


ArQule Announces Orphan Drug Designation in Cholangiocarcinoma and Clinical Update for ARQ 087 [news release]. Burlington, MA; ArQule, Inc.:
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