The FDA Granted Orphan Drug Designation to Biostar Pharma's Utidelone for the Treatment of Pancreatic Cancer

SAN FRANCISCO, Sept. 25, 2025 /PRNewswire/ -- Biostar Pharma, Inc., the US wholly-owned subsidiary of Beijing Biostar Biopharmaceutical Co., Ltd. ("Biostar," stock code: 2563.HK) which is a synthetic biology driven biopharma company focusing on the discovery, development and commercialization of innovative oncology drugs, is pleased to announce that its core pipeline product Utidelone has once again been granted as Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of pancreatic cancer. This marks the third ODD granted to Utidelone by the FDA, following prior designations for breast cancer brain metastases and gastric cancer.

Pancreatic cancer is a highly malignant tumor known as the "king of cancers" due to its insidious early symptoms, difficulty in diagnosis, rapid progression, strong invasiveness, and poor prognosis. The five-year survival rate is only approximately 10%, far lower than that of other common cancers, making it the lowest among all malignant tumors. Currently, there is no clearly effective treatment for pancreatic cancer. Combination regimens based on gemcitabine remain the main clinical treatment, but due to the high tendency of pancreatic cancer cells to develop resistance to gemcitabine, treatment outcomes are often unsatisfactory.

The potential of Utidelone in treating pancreatic cancer has been well validated in both non-clinical and clinical studies^[1]. Preclinical data show that Utidelone can significantly inhibit the proliferation and colony-forming ability of pancreatic cancer cells and exhibits strong anti-tumor activity in pancreatic cancer models. When combined with gemcitabine, Utidelone markedly reduces the IC₅₀ value of gemcitabine without compromising its anti-tumor effect, and the combination demonstrates stronger anti-tumor activity compared with the traditional paclitaxel plus gemcitabine regimen. At the 2024 CSCO Annual Meeting, Biostar presented preliminary results from a multicenter, single-arm Phase II clinical study evaluating Utidelone combined with gemcitabine as first-line treatment for unresectable advanced pancreatic cancer. As of the report date, 20 advanced pancreatic cancer patients which were unresectable and ineligible for local treatment had been enrolled, with 11 completing their first efficacy evaluation. Among them, 3 patients achieved partial response (PR), and 5 achieved stable disease (SD); the objective response rate (ORR) was 27.27%, while the disease control rate (DCR) reached 72.72%. The median overall survival (mOS) was 9.57 months.

About Orphan Drug

An orphan drug is used to treat a rare disease that affects fewer than 200,000 patients in the US. Orphan drug development presents several major challenges including difficulties in patient recruitment, small market size and low return for the pharmaceutical companies. To encourage drug development for the benefit of rare disease patients, an ODD granted by the FDA provides many incentives such as tax relief on clinical trial costs, opportunity to apply for grants to support clinical trials, waiver of new drug application fee, acceleration for regulatory pathway, and the potential to receive 7 years of marketing exclusivity in the US market upon product approval.

About Utidelone

Utidelone is a new-generation genetically engineered microtubule inhibitor, and has similar mechanism of action with that of taxanes while demonstrating multiple advantages, including better anti-tumor activity, broader anti-tumor spectrum, better safety profile with very low hematologic toxicity, effective against multidrug-resistant tumors, less prone to developing drug resistance, capability of crossing the blood-brain barrier to prevent and treat brain tumors, and high oral bioavailability. Biostar has developed two formulations of Utidelone: injection (UTD1) and capsule (UTD2). UTD1 has been launched in China in 2021 for the treatment of metastatic breast cancer (MBC), who have progressed after at least one anthracycline- or taxane-containing chemotherapy regimen. The phase III study data showed that UTD1 plus capecitabine achieved both PFS and OS benefits versus capecitabine for heavily pretreated MBC patients, and the results were orally presented twice at ASCO annual meetings and published in prestigious journals ^[1-2].

About Biostar

Biostar is an integrated biopharma company focusing on the development of innovative anti-cancer drugs utilizing its synthetic biology R&D platform. The company is listed on the Hong Kong Stock Exchange (HK 2563) in 2024. We are actively selecting reliable global partners through out-licensing or co-development of Utidelone assets. We believe that our strong capabilities of R&D and manufacturing, coupled with our enriched commercial expertise, make us the preferred partner for global biopharmaceutical companies who share our goal of bringing innovative anti-cancer products to patients around the world. For additional information on partnering with Biostar, please contact our business development team at [email protected]. 

SOURCE Biostar Pharma, Inc.