Approval and Orphan Drug Exclusivity Granted by FDA For Sodium Oxybate to Treat Cataplexy

The Food and Drug Administration (FDA) has granted approval for an extended-release formulation of the central nervous system depressant sodium oxybate (Lumryz; Avadel Pharmaceuticals, Dublin, Ireland) to treat cataplexy or excessive daytime sleepiness in adults with narcolepsy. This extended-release formulation is meant to be taken at bedtime. According to company statements, product availability is expected in June 2023. In addition, Orphan Drug Exclusivity was granted for the drug beginning in 2023 and ending in 2030 based on data indicating the extended-release formulation to be more effective than currently marketed sodium oxybate products taken twice nightly.   

FDA approval was based on favorable results from the REST-ON phase 3 clinical trial (NCT02720744) which demonstrated significant improvements in participants treated with sodium oxybate 9 g/d compared with placebo in 3 primary endpoints: Maintenance of Wakefulness Test, Clinical Global Impression of Improvement in daytime sleepiness, and mean weekly cataplexy episodes documented on the Sleep and Symptom Daily Diary.

“For people living with narcolepsy, and for all of us who advocate for this community, the approval of LUMRYZ is an important step forward,” said Julie Flygare, JD, President and CEO of Project Sleep.
Common side effects noted with treatment included headache, nausea, vomiting, and enuresis.