Clinical-stage biopharmaceutical company Axsome Therapeutics Inc has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for AXS-12 for the treatment of narcolepsy.AXS-12 is a novel, oral, highly selective and potent norepinephrine reuptake inhibitor. Axsome announced that it plans to initiate a phase 2 trial of AXS-12 for the treatment of the symptoms of narcolepsy in the fourth quarter of this year with topline results anticipated in the first half of 2019.
“We are very pleased to have received Orphan Drug Designation from the FDA for AXS-12 on the heels of our recent announcement of this new CNS [central nervous system] product candidate for the treatment of narcolepsy,” says Herriot Tabuteau, MD, Axsome CEO, in a release. “The designation is an important regulatory milestone in the development of AXS-12 for this debilitating condition. We look forward to starting our planned Phase 2 trial of AXS-12 in patients with narcolepsy this quarter.”
Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to promising drugs intended for the safe and effective treatment of rare diseases, defined as those affecting fewer than 200,000 people in the United States. This designation may entitle Axsome to a period of 7 years of marketing exclusivity in the US upon FDA approval. Orphan Drug Designation also confers special incentives to Axsome including tax credits towards the cost of clinical trials and a waiver of the company’s obligation to pay the FDA application user fees for the product as required by the Prescription Drug User Fee Act.