The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Fujifilm’s FF-10832, an investigational liposomal formulation of gemcitabine, for the treatment of biliary tract cancer (BTC).

Phase 1 study (NCT03440450) results presented at ASCO 2025 suggest FF-10832 is well tolerated and has anti-tumor activity in patients with advanced BTC. FF-10832 is currently being evaluated in phase 2a studies (NCT05318573) as monotherapy or in combination with pembrolizumab for the treatment of solid tumors in the U.S.

Gemcitabine has been a key component of BTC treatment since its approval in the 1990s, with all major first line regimens currently containing gemcitabine. FF-10832’s novel liposomal formulation of gemcitabine for intravenous administration is designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors.

“BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options,” said Susumu Shimoyama, president, FUJIFILM Pharmaceuticals U.S.A., Inc. “Receiving orphan drug designation highlights the significant unmet medical need that still remains and supports development of FF-10832 for patients with BTC who have few satisfactory options.”