FUJIFILM Pharmaceuticals’ FF-10832—an investigational liposomal formulation of gemcitabine—has granted orphan drug designation for the treatment of biliary tract cancer (BTC) by the US Food and Drug Administration (FDA).

FF-10832 is currently being evaluated in Phase IIa studies as monotherapy or in combination with pembrolizumab for the treatment of solid tumours in the US.

Orphan drug 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 US per year. Most patients (≥70%) present with unresectable or metastatic disease at diagnosis.

Current treatments of surgery, chemotherapy, and radiation show limited efficacy in advanced stages. High recurrence (50–70%) and low five-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.

FF-10832’s novel liposomal formulation of gemcitabine for intravenous administration is designed to enhance anti-tumour activity by prolonging plasma half-life and improving targeted delivery to tumours.

“BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options,” said Susumu Shimoyama, President, FUJIFILM Pharmaceuticals USA. “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’s investigational drug candidates FF-10502 and FF-10850 have also been granted orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively.

Diana Turner, Senior Digital Content Editor, DDW