BioXcel's BXCL101, Receives Orphan Drug Designation from the U.S. FDA for the Treatment of Patients with Neurofibromatosis Type 2 (NF2) - EconoTimes

BRANFORD, Conn., Sept. 13, 2016 -- BioXcel, a privately held biopharmaceutical company based in Connecticut, today 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.