XW Laboratories announced the U.S. FDA has granted Orphan Drug Designation to the company’s lead proprietary compound XWL-008 for the treatment of patients with narcolepsy.
The company has successfully completed Phase 1 studies in China, which generated positive safety, tolerability, and pharmacokinetic data. Preparations are underway for late-stage phase 3 clinical research and a 505(b)(2) NDA regulatory pathway as the next step to bringing XWL-008 to narcolepsy patients worldwide.
The FDA Orphan Drug Designation program grants orphan status to promising investigational drugs designed to treat, prevent, or diagnose rare medical diseases or conditions that affect fewer than 200,000 individuals in the United States. Orphan designation qualifies sponsors for several key benefits and incentives, including tax credits for clinical testing, exemption from marketing application user fees, and seven-year marketing exclusivity upon FDA approval.
Narcolepsy is a rare chronic neurological disorder which affects sleep regulation. Loss of neuronal cells called hypocretin leads to abnormal regulation of the sleep-wake cycle. Patients with narcolepsy type 1 typically present with excessive daytime sleepiness (EDS) and cataplexy among other symptoms. Narcolepsy is an orphan disorder that typically affects 1 in 2,000 people.
Most currently available treatments of narcolepsy target one symptom, and those treating both EDS and cataplexy may be associated with intolerable side effects or inconvenient dosing regimen. XWL-008 is a novel agent that is expected not only to be efficacious in treating the primary symptoms of narcolepsy but also improve the tolerability and dosing convenience.Source: