Utidelone Injectable Granted FDA Orphan Drug Designation for Breast Cancer Brain Metastasis


The recent ODD for the utidelone injectable could offer another treatment option for patients with BCBM.

The FDA granted orphan drug designation (ODD) to utidelone injectable (UTD1; Biostar Pharma) to treat breast cancer (BC) brain metastasis (BM).1

Doctor examines mammogram snapshot of breast of female patient on the monitors. Selective focus. - Image credit: okrasiuk | stock.adobe.com

Image credit: okrasiuk | stock.adobe.com

BM occurs when cancer cells break away from the primary tumor and travel to the brain, most commonly through the blood stream, study authors noted. However, in some cases it could take many years for BM to appear following the primary cancer diagnosis. Common signs and symptoms of brain metastases include headaches, seizures, weakness in the arms or legs, loss of balance, memory loss, speech disturbance, behavior and personality changes, blurred vision, numbness, and hearing loss.2

Breast cancer is among the most common types of cancer that can spread to the brain, occurring in 30% of metastatic breast cancer diagnoses, according to study authors. Research has displayed that metastatic brain tumors are 5 times more common than primary brain tumors and can grow rapidly. Individuals that develop brain metastases (BM) often experience a low survival rate and the survival duration of BCBM is a median of 7.2 months. However, the study authors noted that the survival rate could be only 3.5 months if diagnosed with triple-negative breast cancer BM.1,2

The study authors explained further that there is low therapeutic efficacy to treat BCBM because macromolecular drugs are not able to penetrate the blood-brain barrier (BBB), with surgery and radiation as the standard form of treatment. Currently, there is 1 treatment for BCBM for individuals diagnosed globally, suggesting the need for further treatments to aid patients.1

However, utidelone could offer another treatment option for patients as it can cross BBB because of its “physiochemical characteristic and insusceptibility to P-glycoprotein-mediated efflux,” according to study authors.1

The results from the phase 2 clinical trial assessing utidelone injectable with etoposide and bevacizumab for HER2 negative BCBM individuals (n=17) displayed excellent central nervous system (CNS) overall response rate (ORR) and CNS clinical benefit rate (CBR) of 73% and 91%, respectively. Results from a separate phase 2 clinical trial that evaluated utidelone injectable in combination with bevacizumab for HER2 negative BCBM patients (n=46) also demonstrated positive outcomes of excellent CNS overall response rate (ORR) and CNS clinical benefit rate (CBR) of 73% and 91%, respectively.1

“Biostar Pharma also plans to advance the clinical studies of utidelone injectable for the treatments of other brain tumors such as lung cancer brain metastasis and glioma this year,” said study authors in the press release.1

1. 73% CNS ORR! FDA Granted ODD to Utidelone Injectable (UTD1) from Biostar Pharma for the Treatment of Breast Cancer Brain Metastasis. PR Newswire. News release. March 29, 2024. Accessed March 29, 2024. 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
2. Johns Hopkins Medicine. Metastatic Brain Tumors. Accessed March 29, 2024. https://www.hopkinsmedicine.org/health/conditions-and-diseases/metastatic-brain-tumors.
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