Avadel Pharmaceuticals plc has been granted Orphan Drug Designation from the US Food and Drug Administration (FDA) for FT 218 for the treatment of narcolepsy.
FT 218 is a once-nightly formulation of sodium oxybate using Avadel’s proprietary Micropump technology. It is currently undergoing testing in a Phase III clinical trial for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients suffering from narcolepsy. The designation has been granted on the plausible hypothesis that FT 218 may be clinically superior to the same drug already approved for the same indication because FT 218 may be safer due to ramifications associated with the dosing regimen of the previously-approved product.
Mike Anderson, Avadel’s CEO, says in a release, “Receipt of Orphan Drug Designation for FT 218 is meaningful for both Avadel and patients suffering from narcolepsy. Narcolepsy is a debilitating and rare sleep disorder for which limited treatment options exist. We look forward to completing our REST-ON Phase III trial this year and are hopeful that FT 218 can provide meaningful benefit to patients and their quality of life over other standards of care.”
Orphan Drug status is intended to advance drug development for rare diseases. The FDA provides Orphan Drug Designation to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a 7-year period of market exclusivity in the United States after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
REST-ON is a double-blind, randomized, placebo controlled study of 264 patients to assess the efficacy and safety of a once nightly formulation of sodium oxybate for extended-release oral suspension for the treatment of excessive daytime sleepiness and cataplexy in patients suffering from narcolepsy.