Biostar Pharma, the U.S. subsidiary of Beijing Biostar Biopharmaceutical, announced that its core drug candidate, Utidelone, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer. This marks the third ODD for Utidelone, following earlier designations for breast cancer brain metastases and gastric cancer.
Pancreatic cancer, often called the “king of cancers,” is highly aggressive with a poor prognosis and a five-year survival rate of approximately 10%, the lowest among malignant tumors. Its early symptoms are subtle, diagnosis is challenging, and treatment options remain limited. The current standard treatment typically involves gemcitabine-based regimens, but resistance to gemcitabine frequently leads to unsatisfactory outcomes.
Utidelone’s potential against pancreatic cancer has been demonstrated in preclinical and clinical studies. Laboratory data indicate that Utidelone significantly inhibits pancreatic cancer cell growth and tumor formation. When combined with gemcitabine, Utidelone enhances the chemotherapy’s effectiveness without diminishing its anti-tumor properties. This combination showed stronger anti-tumor activity compared to the conventional paclitaxel and gemcitabine treatment.
At the 2024 Chinese Society of Clinical Oncology (CSCO) Annual Meeting, Biostar presented preliminary results from a multicenter Phase II clinical trial testing Utidelone combined with gemcitabine as a first-line therapy for unresectable advanced pancreatic cancer. Out of 20 enrolled patients, 11 had completed their first efficacy evaluation, with three patients achieving partial response and five showing stable disease. The objective response rate stood at 27.27%, with a disease control rate of 72.72%, and a median overall survival of 9.57 months.
This FDA designation highlights Utidelone’s promise as a new therapeutic option in the fight against pancreatic cancer.