Core Points - BioArctic AB has received orphan drug designation from the US FDA for exidavnemab, aimed at treating Multiple System Atrophy (MSA), a rare and fatal disease with no current cure [1][3][7] - Exidavnemab is a monoclonal antibody designed to target and eliminate aggregated alpha-synuclein, potentially slowing the progression of MSA and Parkinson's disease [2][8] - The orphan drug designation provides various incentives, including tax credits for clinical trials and potential seven-year marketing exclusivity upon FDA approval [3] Company Overview - BioArctic AB is a Swedish biopharma company focused on innovative treatments for neurodegenerative diseases, including Alzheimer's and Parkinson's [9] - The company is known for developing Leqembi® (lecanemab), the first drug proven to slow Alzheimer's progression [9] - BioArctic utilizes proprietary technology, such as BrainTransporter™, to enhance drug delivery to the brain [9] Disease Context - MSA is characterized by rapid progression and affects the central and autonomic nervous systems, leading to severe disability and a poor prognosis [6][7] - The disease affects fewer than 42,000 individuals in the U.S., highlighting the high unmet medical need [7] - Patients typically have a life expectancy of 6 to 10 years post-symptom onset, with few surviving beyond 15 years [7]
BioArctic receives Orphan Drug Designation for exidavnemab the US