BioXcel, a privately held biopharmaceutical company based in Connecticut, announced that the U. S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BXCL101 for the treatment of Neurofibromatosis Type 2 (NF2), an orphan disease with significant unmet medical need. BXCL101 is the first and only systemic therapy being developed to eliminate existing lesions and prevent the formation of new lesions by targeting the molecular mechanism of NF2 pathophysiology.
BXCL101 is a proprietary version of an approved drug, bortezomib, adapted for chronic use in NF2 patients with both a novel dosing regimen and delivery approach. NF2 is a rare disease associated with neurologic and ophthalmologic abnormalities caused by benign tumors of the brain, spinal cord and peripheral nerves. BXCL101 is the first drug candidate discovered using BioXcel’s R&D Platform, to be granted orphan drug status by the FDA. BioXcel is advancing the clinical development of BXCL101 and is exploring global partnering opportunities.
“Securing Orphan Drug Designation from the FDA’s Office of Orphan Products Development (OOPD) is an important milestone for BioXcel. Moreover, it provides a crucial regulatory validation of our business model, to identify clinical candidates that address areas of high unmet medical need and bring them quickly to confirmatory clinical trials,” said Dr. Vimal D. Mehta, CEO and Chairman of BioXcel.
“BXCL101 represents a unique solution to address this rare and debilitating disease, where the current standard of care is not sufficient to satisfy patient needs,” added Dr. Krishnan Nandabalan, President and CSO of BioXcel. “We believe that BXCL101 has the potential to significantly improve the lives of patients with NF2 and we look forward to advancing its development, as well as further growing our therapeutic pipeline with additional drug candidates.”
