- The FDA has granted orphan drug designation to FF-10832 (liposomal gemcitabine) in biliary tract cancer (BTC), highlighting the significant unmet medical need for this aggressive and rare malignancy.
- Liposomal gemcitabine is an investigational formulation designed to enhance antitumor activity by prolonging plasma half-life and improving targeted drug delivery, potentially offering a more effective treatment option.
- This designation provides crucial incentives for the development of FF-10832, including market exclusivity and financial support, accelerating its path through clinical trials for a disease with limited current therapies.
The FDA has granted orphan drug designation to FF-10832, an investigational liposomal formulation of gemcitabine, for the treatment of BTC. This designation underscores the critical unmet medical need in BTC, a rare and aggressive malignancy with poor prognosis and limited therapeutic options.1
BTCs, including cholangiocarcinoma and gallbladder cancer, affect approximately 16,000 individuals annually in the US. Over 70% of patients present with unresectable or metastatic disease at diagnosis, contributing to high recurrence rates of 50% to 70% and 5-year survival rates ranging from 4% to 13%. Despite its approval in the 1990s, gemcitabine remains a cornerstone of first-line BTC treatment regimens, highlighting the persistent demand for more effective interventions.
FF-10832 represents a novel approach to delivering gemcitabine, a widely used antimetabolite. The liposomal formulation is designed for intravenous administration and aims to enhance antitumor activity by prolonging plasma half-life and improving targeted delivery of the drug to tumor sites. This innovative delivery system holds the potential to improve the therapeutic index of gemcitabine, leading to greater efficacy and potentially reduced systemic toxicity compared with conventional formulations.
The investigational agent is currently undergoing evaluation in phase 2a clinical studies (NCT05318573). These trials are assessing the safety and efficacy of liposomal gemcitabine as monotherapy and in combination with pembrolizumab (Keytruda), an immune checkpoint inhibitor, across various solid tumors, including BTC.
Microscopic, photorealistic image of tumor cells – Generated with Adobe Firefly
The FDA’s orphan drug designation is specifically aimed at facilitating the development of therapies for rare diseases impacting fewer than 200,000 people in the US. This designation provides significant incentives, including a 7-year period of marketing exclusivity upon approval, along with tax credits for clinical research costs and exemption from FDA user fees.2 These provisions are crucial for advancing research and clinical development in patient populations where small numbers can impede preclinical research and slow down clinical trials, thereby hindering therapeutic progress.
“BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options,” said Susumu Shimoyama, president of Fujifilm Pharmaceuticals, in a press release.1 “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.”
Fujifilm Pharmaceuticals’ investigational drug candidates FF-10502 and FF-10850 have also received orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively. The potential for liposomal gemcitabine to improve outcomes in BTC patients warrants continued close observation as clinical trial data emerges. The strategic benefit of enhanced drug delivery through liposomal encapsulation could offer a much-needed advancement for a disease area that has seen limited progress in recent decades.