Sumitomo Pharma Oncology, a clinical-stage company focused on novel cancer therapeutics, announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for DSP-0390, an investigational emopamil-binding protein (EBP) inhibitor for the treatment of brain cancer.

"This designation for DSP-0390 underscores the profound need for novel brain cancer treatment options," said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. "We are excited to contribute to advancing critical research in brain cancer." 

The FDA's Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Brain cancer refers to brain tumors, made up of abnormal growth of cells in the brain, that are malignant. The exact cause of most brain cancer is unknown.

"DSP-0390 is an emopamil-binding protein (EBP) inhibitor that mediates de novo cholesterol synthesis for cell membrane structure and signaling, enabling aberrant growth of tumors,"2-5 detailed Jatin J. Shah, M.D., Chief Medical Officer of Sumitomo Pharma Oncology, Inc.

"Notably, in patients with glioblastoma multiforme (GBM), a form of high-grade glioma, increased de novo cholesterol synthesis is correlated with poor survival and preclinical evidence has shown that DSP-0390 has cytotoxic activity against GBM cell lines."

DSP-0390 is currently being evaluated in a Phase 1 clinical trial to evaluate the safety and efficacy of DSP-0390 in patients with recurrent, high-grade glioma, which is being conducted in the United States and Japan.