FDA Gives Brain Metastasis Drug Orphan Drug Designation


The Food and Drug Administration granted UTD1 an orphan drug designation to treat brain metastases in patients with breast cancer.

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The novel UTD1 treatment received an orphan drug designation by the FDA for patients with breast cancer who have brain metastases.

The Food and Drug Administration (FDA) granted an orphan drug designation to utidelone injectable (UTD1) for the treatment of brain metastases in patients with breast cancer, according to a press release from Biostar Pharmaceuticals, the manufacturer of the drug.

The agency grants orphan drug designations to drugs that show potential in treating a rare disease. The designation comes with trial tax credits, fee exemptions and the potential of seven years of market exclusivity, per the FDA.

An Unmet Need for Brain Metastases

New therapies are needed for breast cancer that has spread to the brain, as long-term survival for this patient population is poor, according to Biostar. Approximately 30% of patients with metastatic breast cancer will have disease in the brain. The median survival for this group is approximately 7.2 months and 3.5 months for patients with triple-negative disease.

Brain metastases are difficult to treat because most therapies cannot cross the blood-brain barrier, which is a membrane that regulates what kind of molecules can pass into the brain and central nervous system, according to the National Institutes of Health.

READ MORE: Educated Patient® Breast Cancer Summit at MBCC Treating Brain Metastases

UTD1 Crosses the Blood-Brain Barrier

UTD1, however, has demonstrated the ability to cross the blood-brain barrier.

The small phase 2 UTOBIA-BM trial of UTD1 plus etoposide and Avastin (bevacizumab) showed that 73% of patients with HER2-negative breast cancer brain metastases responded to the therapy. Further, 91% experienced a central nervous system clinical benefit. At the last time the researchers collected data, not enough patients experienced disease progression or death to calculate an average time until either of those events.

Patients in the trial were all female and had a life expectancy of 12 weeks or longer. All patients had prior HER2-targeted therapy and TKI therapy.

Another phase 2 trial of UTD1 with Avastin in 46 patients with HER2-negative breast cancer with brain metastases showed that patients lived for a median of 7.7 months before their disease got worse. At 12 months, 74.4% of patients were still alive. Now, Biostar plans on studying UTD1 in other diseases as well.

“Considering [UTD1’s] excellent [blood-brain barrier]-crossing capability and its therapeutic potential for brain tumors, Biostar Pharma also plans to advance the clinical studies of [UTD1] injectable for the treatments of other brain tumors such as lung cancer brain metastasis and glioma this year,” the company said in the press release.

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