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FDA grants orphan drug designation to ARQ 087 for cholangiocarcinoma
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The FDA granted orphan drug designation to ARQ 087 for the treatment of patients with cholangiocarcinoma, according to a press release from the drug’s manufacturer.
ARQ 087 (ArQule) — a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth receptor (FGFR) family — is currently under investigation in a phase 2 trial conducted in the U.S. and Italy. The manufacture plans to enroll 20 patients with intrahepatic cholangiocarcinoma and FGFR2 fusions in the study, according to the release.
In the phase 1a portion of the study, two patients with intrahepatic cholangiocarcinoma and FGFR2 fusions achieved partial responses with ARQ 087, and a third patient achieved a minor response.
“[Cholangiocarcinoma] is a rare cancer that lacks approved therapeutic options to specifically target FGFR2 fusions,” Brian Schwartz, MD, chief medical officer at ArQule, said in the release. “We are very encouraged by the clinical efficacy so far observed in the phase 1 portion of the trial which validates the preclinical data recently presented at the AACR-NCI-EORTC conference that demonstrated efficacy across a number of FGFR2-driven tumors in xenograft models.”
ARQ 087 is also under investigation in the phase 1b portion of the trial, composed of patients with solid tumors harboring FGFR2 genetic alterations. One patient with FGFR2-mutated bladder cancer has achieved a partial response.