Swedish research-based biopharma company BioArctic AB (STO:BIOA-B) announced on Monday that it has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for exidavnemab for the treatment of Multiple System Atrophy (MSA), a rapidly progressive and fatal rare disease affecting the central and autonomic nervous systems.

Exidavnemab is being developed as a novel disease-modifying treatment for synucleinopathies such as Multiple System Atrophy (MSA) and Parkinson's disease. It is a monoclonal antibody (mAb) that selectively targets soluble alpha-synuclein aggregates, such as oligomers or protofibrils.

The FDA's Orphan Drug Designation programme provides orphan status to drugs or biologics intended for the treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives and regulatory assistance, including tax credits for qualified clinical trials, prescription drug user-fee exemptions and potential seven-year marketing exclusivity upon FDA approval.