Bexion Wins Orphan Status for Pediatric Glioma Drug

FDA has granted Bexion Pharmaceuticals an orphan drug designation for BXQ-350 and its use for treating malignant glioma, including diffuse intrinsic pontine glioma (DIPG), which primarily affects children. The company says DIPG makes up 10-15% of all brain tumors in children, with about 150-300 new diagnoses per year in the U.S. It describes the therapy as a first-in-class agent composed of the multifunctional, lysosomal activator protein Saposin C and phosphatidylserine.

“BXQ-350 has demonstrated pre-clinical antitumor effects in vitro and in vivo, particularly in brain and other solid tumors, including those that may lead to brain metastases,” Bexion says, adding that it has completed a multi-site first-in-human Phase 1 clinical trial of BXQ-350 for solid tumors and gliomas, and a Phase 1 pediatric trial completed enrollment in late 2019.