FF-10832 Granted FDA Orphan Drug Designation for the Treatment of Biliary Tract Cancer

CAMBRIDGE, Mass.--(BUSINESS WIRE)--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, today announced 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). Phase 1 study (NCT03440450) results presented at ASCO 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.

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.

About FUJIFILM

FUJIFILM Pharmaceuticals U.S.A., Inc., based in Boston, Massachusetts, specializes in next generation drug delivery system formulation and CDMO services for precision treatment and care. In addition, the Company builds and maintains relationships with medical institutions, academic partners, and industry partners to conduct clinical trials and advance development of unique therapeutic compounds in oncology pipelines. For more information, please visit www.fujifilmpharma.com.

FUJIFILM Corporation is a subsidiary of FUJIFILM Holdings Corporation. FUJIFILM Holdings Corporation, headquartered in Tokyo, leverages its depth of knowledge and proprietary core technologies to deliver innovative products and services across the globe through the four key business segments of healthcare, electronics, business innovation, and imaging with over 70,000 employees. Guided and united by our Group Purpose of “giving our world more smiles,” we address social challenges and create a positive impact on society through our products, services, and business operations. Under its medium-term management plan, VISION2030, which ends in FY2030, we aspire to continue our evolution into a company that creates value and smiles for various stakeholders as a collection of global leading businesses and achieve a global revenue of 4 trillion yen (29 billion USD at an exchange rate of 140 JPY/USD). For more information, please visit: www.fujifilmholdings.com.

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^1 Food and Drug Administration (FDA). 2013. 21CFR Part 316 - Relevant Excerpts. Available at https://www.fda.gov/industry/designating-orphan-product-drugs-and-biological-products/orphan-drug-act-relevant-excerpts, accessed 2025 May 10.

^2 Lowe RC, Anderson CD. 2024. Epidemiology, risk factors, anatomy, and pathology of cholangiocarcinoma. UpToDate [website]. Available at: https://www.uptodate.com/contents/epidemiology-risk-factors-anatomy-and-pathology-of-cholangiocarcinoma?search=cholangiocarcinoma&source=search_result&selectedTitle=4%7E145&usage_type=default&display_rank=4, accessed 2025 April 29.

^3 Rizzo A, Brandi G. 2021. Cancer Treat Res Commun 27:100335, PMID: 33592561.