FDA Grants Orphan Drug Designation to Utidelone in Pancreatic Cancer

The U.S. Food and Drug Administration (FDA) has reportedly granted orphan drug designation to utidelone for the treatment of pancreatic cancer.

The development was announced in a news release issued by Biostar Pharma, a U.S. subsidiary of Beijing Biostar Pharmaceutical Co., Ltd., a synthetic biology-driven biopharma company. Utidelone, which is Biostar’s core pipeline product, previously received orphan drug designation from the FDA for the treatment of breast cancer brain metastases and gastric cancer, according to the news release.

The FDA, the agency explains on its website, can grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition, with orphan drug designation qualifying sponsors for incentives such as tax credits for qualified clinical trials, exemptions from user fees and a potential seven years of market exclusivity following FDA approval.

Utidelone, according to the news release, is a treatment described as a new-generation genetically engineered microtubule inhibitor, and it is said to have similar mechanism of action to taxanes while demonstrating multiple advantages, such as better anti-tumor activity, broader anti-tumor spectrum, a better safety profile with very low hematologic toxicity, and is effective against multidrug-resistant tumors, less prone to developing drug resistance, capable of crossing the blood-brain barrier to prevent and treat brain tumors, and high oral bioavailability. Biostar has developed both injectable and capsule forms of utidelone, known as UTD1 and UTD2, respectively.

The news release further stated that utidelone’s usefulness in potentially treating pancreatic cancer has already been demonstrated in both non-clinical and clinical studies, with preclinical data being said to show that the drug can significantly inhibit the proliferation and colony-forming ability of pancreatic cancer cells and exhibit anti-tumor activity in pancreatic cancer models.

Clinical Trial Findings for Utidelone in Pancreatic Cancer

At last year’s CSCO Annual Meeting, preliminary data from a phase 2 clinical study of utidelone plus gemcitabine as a first-line treatment for advanced pancreatic cancer that was unresectable, or unable to be removed with surgery, showed that among 11 patients who had completed their first efficacy evaluation three patients had achieved a partial response to treatment and five patients had achieved stable disease, with an objective response rate of 27.27%, a disease control rate of 72.72% and a median overall survival, or the time a patient lives, of 9.57 months, according to the news release.

Regarding side effects, Biostar has reported that in the phase 2 trial most of the side effects were grade 1 (mild) or grade 2 (moderate), with only one patient having a grade 3 (severe) to 4 (life-threatening) side effect of granulocytopenia or a low level of granulocytes, a type of white blood cell. This patient, the company stated, recovered after dose adjustment, which is an indication that the treatment was generally safe and controllable.

Pancreatic Cancer Risk and Survival Rate Statistics

In 2025 in the United States, approximately 67,440 people, 34,950 men and 32,490 women, will receive a diagnosis of pancreatic cancer, and approximately 51,980 people, 27,050 men and 24,930 women, will die of the disease, according to statistics published by the American Cancer Society on its website.

The average lifetime risk of pancreatic cancer is approximately one in 56 men and one in 60 women, while, based on patients who received a diagnosis of pancreatic cancer between 2014 and 2020, the disease’s five-year relative survival rate is just 13%, according to the American Cancer Society.

References

1. “The FDA Granted Orphan Drug Designation to Biostar Pharma’s Utidelone for the Treatment of Pancreatic Cancer,” news release; https://www.biostar-pharm.com/en/news/view/269.html
2. “FDA Gives Brain Metastasis Drug Orphan Drug Designation,” CURE, April 2, 2024; https://www.curetoday.com/view/fda-gives-brain-metastasis-drug-orphan-drug-designation
3. “Designating an Orphan Product: Drugs and Biological Products,” FDA; https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
4. “Preliminary data released from a clinical study of Utidelone for the first-line treatment of advanced pancreatic cancer,” news release; https://www.biostar-pharm.com/en/news/view/210.html
5. “About Pancreatic Cancer,” American Cancer Society; https://www.cancer.org/cancer/types/pancreatic-cancer/about.html

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