FDA Approves Orphan Drug Status for Liposomal Gemcitabine in Biliary Tract Cancer

On July 8, 2025, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to FF-10832, an investigational liposomal formulation of gemcitabine, for the treatment of biliary tract cancer (BTC). This designation highlights the significant unmet medical need associated with this rare and aggressive malignancy, which is characterized by poor prognosis and limited treatment options.

Biliary tract cancer, which includes cholangiocarcinoma and gallbladder cancer, affects approximately 16,000 individuals annually in the United States. According to the American Cancer Society, over 70% of patients present with unresectable or metastatic disease at diagnosis, leading to high recurrence rates of 50% to 70% and significantly low five-year survival rates ranging from 4% to 13% (American Cancer Society, 2023).

Gemcitabine, a widely used chemotherapeutic agent, has been a cornerstone of first-line treatment regimens for BTC since its approval in the 1990s. However, the persistent demand for more effective interventions has prompted the development of FF-10832, which aims to enhance the drug's antitumor activity by prolonging plasma half-life and improving targeted drug delivery to tumor sites (Fujifilm Pharmaceuticals, 2025).

FF-10832's liposomal formulation is designed for intravenous administration, potentially improving the therapeutic index of gemcitabine and reducing systemic toxicity compared to conventional formulations. Currently, it is being evaluated in Phase 2a clinical trials (NCT05318573), assessing its safety and efficacy as a monotherapy and in combination with pembrolizumab (Keytruda), an immune checkpoint inhibitor, across various solid tumors, including BTC (ClinicalTrials.gov, 2025).

The FDA’s orphan drug designation not only facilitates the development of therapies for rare diseases, but also offers significant incentives such as a seven-year period of marketing exclusivity upon approval, tax credits for clinical research costs, and exemption from FDA user fees (FDA, 2024). These provisions are vital for advancing research and clinical development in patient populations where small numbers can impede preclinical studies and slow down clinical trials, thereby hindering therapeutic progress.

“Biliary tract cancers are rare but aggressive malignancies associated with a poor prognosis and limited treatment options,” stated Susumu Shimoyama, president of Fujifilm Pharmaceuticals. “Receiving orphan drug designation highlights the significant unmet medical need that still remains and supports the development of FF-10832 for patients with BTC who have few satisfactory options” (Fujifilm Pharmaceuticals, 2025).

The potential for liposomal gemcitabine to improve outcomes in BTC patients represents a significant advancement in a field that has seen limited progress over the past few decades. As clinical trial data emerges, the medical community will closely monitor the efficacy and safety of this innovative treatment option, which could change the landscape of care for patients suffering from this challenging disease.

In summary, the FDA's orphan drug designation of FF-10832 underscores the urgent need for new therapeutic strategies in biliary tract cancer, offering hope for patients and healthcare providers alike in the ongoing fight against this aggressive malignancy.