Bexion Pharmaceuticals, Inc. issued the following announcement on May 7.
Bexion Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted the company Orphan Drug Designation for its proprietary drug, BXQ-350, for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG). DIPG primarily affects children, with most diagnoses occurring between 5 and 7 years of age. It makes up 10-15% of all brain tumors in children, with about 150-300 new diagnoses per year in the United States. Unfortunately, fewer than 10% of children survive two years from diagnosis.
The FDA's Office of Orphan Drug Products Development reviews applications for Orphan Drug Designation to support development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the United States. The successful application submitted by Bexion and the FDA granting of Orphan Drug status entitles the company to a seven-year period of marketing exclusivity in the United States for BXQ-350 upon approval by the FDA for the treatment of malignant gliomas, including DIPG. Orphan Drug status also enables the company to apply for research grant funding for Phase I and II Clinical Trials, tax credits for certain research expenses, a waiver from the FDA's application user fee, as well as additional support from FDA and a potentially faster regulatory process.
"Receiving this orphan drug designation for BXQ-350 is another important milestone emerging from our innovative technology to treat multiple cancer types," said Ray Takigiku, PhD; CEO and President of Bexion Pharmaceuticals. "We are working to enter advanced trials of BXQ-350 this year and hope to define new and improved treatment options."
About Bexion Pharmaceuticals
Bexion Pharmaceuticals is a clinical-stage biopharmaceutical company developing BXQ-350, 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 has completed a multi-site first-in-human Phase 1 clinical trial of BXQ-350 for solid tumors and gliomas. A Phase 1 Pediatric Trial enrollment was completed in late 2019.
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