Bexion’s Saposin C – the active ingredient in the brain tumour therapy BXQ-350 – has been awarded Orphan Drug status by US regulators.
The company is seeking approval to market its drug for glioblastoma multiforme, the most common and most aggressive malignant primary brain tumour in humans.
Orphan Drug status, which can be applied to experimental medicines for underserved patient populations or rare disorders affecting less than 200,000 people, entitles the company to a seven-year period of marketing exclusivity in the US following approval.
“Few treatment options are available for patients suffering from glioblastoma multiforme and this designation recognizes the unmet need that exists with this disease, as well as the unique attributes of BXQ-350,” noted founder and chief executive Ray Takigiku.
It also also enables the company to benefit from important financial, regulatory and commercial considerations, including tax credits for certain research expenses and a waiver from the FDA’s application user fee, he said.
