FDA Grants Utidelone Injection Orphan Drug Designation for Pancreatic Cancer

The FDA has granted orphan drug designation to utidelone injection (UTD1) for the treatment of patients with pancreatic cancer, according to an announcement from Biostar Pharma.1 Of note, utidelone has previously received orphan drug designation for the treatment of patients with breast cancer brain metastases and gastric cancer.

In April 2024, the FDA granted orphan drug designation to utidelone injection as a potential therapeutic option for the treatment of patients with breast cancer with brain metastases.2 The regulatory decision was based on data from 2 phase 2 trials.

The orphan drug designation for utidelone in pancreatic cancer has been validated in nonclinical and clinical studies.1 Specifically, preclinical data demonstrated that utidelone can inhibit the proliferation of pancreatic cancer cells and shows strong antitumor activity among pancreatic cancer models. Additionally, when combined with gemcitabine, utidelone demonstrated a reduction in the IC50 value of gemcitabine without compromising the antitumor effect. Notably, the combination showed strong antitumor activity compared with the traditional combination of paclitaxel and gemcitabine as first-line treatment of patients with unresectable advanced pancreatic cancer.

At the 2024 Chinese Society of Clinical Oncology Annual Meeting, preliminary results from a multicenter, single-arm phase 2 trial (NCT05795920) revealed that 20 patients with unresectable advanced pancreatic cancer treated with first-line utidelone plus gemcitabine who were ineligible to receive local treatment were enrolled on the study. Eleven patients from the study have completed their first efficacy evaluation, of which 3 patients achieved partial response and 5 achieved stable disease with the combination. The objective response rate with utidelone plus gemcitabine was 27.27%, the disease control rate was 72.72%, and the median overall survival was 9.57 months.

What is the Design of the Phase 2 Study?

Patients included on the study were between 18 years of age and 75 years of age, had a Karnofsky physical status score of at least 70, and had histopathological or cytological inoperable, stage III to IV pancreatic cancer that was unsuitable for local treatment.3 Furthermore, patients were required to have a baseline blood routine and biochemical indexes, which included hemoglobin of at least 90 g/L, neutral Granulocyte absolute count of at least 1.5 x 109 g/L, platelet count of at least 100 x 109/L, alanine aminotransferase and aspartate aminotransferase levels of less than 2.5 times the normal upper limit, serum total bilirubin levels of less than 1.5 times the normal upper limit, serum creatinine levels of less than 1 times the upper normal limit, and serum albumin levels of at least 30 g/L.

Patients were not permitted to enroll on the study if they had previously received chemotherapy, radiotherapy, or immunotherapy for the treatment of pancreatic cancer, had histopathologically or cytologically confirmed pancreatic neuroendocrine tumors, and had a history of uncontrolled epilepsy, central nervous system disease or mental disorder. Moreover, patients with clinically severe and active heart disease, including systemic coronary heart disease, and severe uncontrolled recurrent infection or other severe uncontrolled concomitant disease were not included on the study.

Patients enrolled on the study were treated with utidelone injection plus gemcitabine. In particular, utidelone was given once daily at 30 mg/m2 on days 1 to 5 for a 21-day treatment cycle. Gemcitabine was given once every 3 weeks at 1000 mg/m2 on days 1 and 8 in the 21-day treatment cycle.

The primary end points of the study included overall survival and progression-free survival. The secondary end point was duration of response.

References

1. The FDA granted orphan drug designation to Biostar Pharma’s utidelone for the treatment of pancreatic cancer. News release. Biostar Pharma. September 25, 2025. Accessed September 25, 2025. https://www.biostar-pharm.com/en/news/view/269.html
2. 73% CNS ORR! FDA granted ODD to utidelone injectable (UTD1) from Biostar Pharma for the treatment of breast cancer brain metastases. News release. Biostar Pharma. March 29, 2024. Accessed September 25, 2025. https://www.prnewswire.com/news-releases/73-cns-orr-fda-granted-odd-to-utidelone-injectable-utd1-from-biostar-pharma-for-the-treatment-of-breast-cancer-brain-metastasis-302103497.html
3. Utidelone injection combined with gemcitabine in first-line chemotherapy for unresectable advanced pancreatic cancer. ClinicalTrials.gov. Updated April 3, 2023. Accessed September 25, 2025. https://clinicaltrials.gov/study/NCT05795920