FUJIFILM Pharmaceuticals U.S.A., Inc. announced that the U.S. Food and Drug Administration has granted orphan drug designation to FF-10832, an investigational liposomal formulation of gemcitabine, for the treatment of biliary tract cancer (BTC). The designation provides seven years of marketing exclusivity and financial incentives to support development of therapies for rare diseases affecting fewer than 200,000 people in the United States.
Clinical Development Progress
Phase 1 study results (NCT03440450) presented at ASCO 2025 demonstrated that FF-10832 is well tolerated and exhibits anti-tumor activity in patients with advanced biliary tract cancer. The drug is currently being evaluated in phase 2a studies (NCT05318573) both as monotherapy and in combination with pembrolizumab for treating solid tumors in the United States.
Addressing Significant Unmet Medical Need
Biliary tract cancer represents a substantial clinical challenge, with approximately 16,000 new cases diagnosed annually in the United States. The disease presents particular treatment difficulties, as most patients (≥70%) are diagnosed with unresectable or metastatic disease. Current treatment options including surgery, chemotherapy, and radiation demonstrate limited efficacy in advanced stages of the disease.
The prognosis for biliary tract cancer patients remains poor, with high recurrence rates ranging from 50-70% and low five-year survival rates of only 4-13%. These statistics underscore the urgent need for new and effective therapeutic approaches.
Enhanced Drug Delivery Technology
FF-10832's novel liposomal formulation represents an advancement over conventional gemcitabine delivery. The intravenous formulation is specifically designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors. Gemcitabine has been a cornerstone of biliary tract cancer treatment since its approval in the 1990s, with all major first-line treatment regimens currently incorporating the drug.
"BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options," said Susumu Shimoyama, president of 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."
Manufacturing and Development Pipeline
FF-10832 is manufactured by FUJIFILM Toyama Chemical, which provides comprehensive contract development and manufacturing organization services with full integration from formulation development to GMP manufacturing. The orphan drug designation for FF-10832 adds to FUJIFILM's growing oncology pipeline, joining other investigational drug candidates FF-10502 and FF-10850, which have received orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively.
