On July 7, 2025 FUJIFILM Pharmaceuticals U.S.A., Inc., drug development center and a leading provider of Contract Development and Manufacturing Organization (CDMO) services for drug delivery system (DDS) technologies, reported that 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) (Press release, Fujifilm, JUL 7, 2025, View Source [SID1234654270]). Phase 1 study (NCT03440450) results presented at ASCO (Free ASCO Whitepaper) 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.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
The FDA grants orphan drug designation to support the development of therapies for rare diseases that affect fewer than 200,000 people in the U.S.1 Small patient populations can limit preclinical research and slow clinical trials, stalling progress in therapeutic development for rare diseases. The designation supports research and clinical development by providing 7-years marketing exclusivity and other financial incentives.
There are approximately 16,000 new cases of BTC in the U.S per year.2 Most (≥70%) patients present with unresectable or metastatic disease at diagnosis, and current treatments of surgery, chemotherapy, and radiation show limited efficacy in advanced stages. High recurrence (50–70%) and low 5-year survival rates (4–13%) highlight the urgent need for new and effective treatments.
Gemcitabine has been a key component of BTC treatment since its approval in the 1990s, with all major first line regimens currently containing gemcitabine.3 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."
FF-10832 is manufactured by FUJIFILM Toyama Chemical, providing seamless CDMO end-to-end services for full integration from formulation development to GMP manufacturing. Fujifilm’s investigational drug candidates FF-10502 and FF-10850 have also been granted orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively.