The US Food and Drug Administration (FDA) has granted tentative approval to LUMRYZ, also known as FT218, by Avadel Pharmaceuticals plc. 

The FDA grants “tentative” approval while delaying final approval of drugs until all patent or exclusivity issues have been resolved. A tentative approval does not allow the applicant to market the generic drug product. 

Tentative approval indicates that LUMRYZ has met all required quality, safety, and efficacy standards necessary for approval in the United States. In this case, final approval is pending disposition of US patent No. 8,731,963 (the “REMS patent”), which is listed in FDA’s Orange Book. 

LUMRYZ is a once-at-bedtime investigational formulation of sodium oxybate for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy.

“We have reached a critical milestone, as tentative approval confirms the safety profile and clinical efficacy of LUMRYZ for adults with narcolepsy,” says Greg Divis, CEO at Avadel Pharmaceuticals, in a release. “Tentative approval is an important regulatory step forward and indicates LUMRYZ could potentially be granted final approval in 11 months or less. We believe once-at-bedtime LUMRYZ offers the opportunity to positively transform the lives of oxybate eligible patients living with narcolepsy. Our extensive market research indicates Avadel is well-positioned to capture significant share of the oxybate eligible patient population, which we estimate to be in excess of 30,000 patients. We are pursuing all options to accelerate final approval on or before June 2023 and prepare for commercial launch.”

With tentative approval now secured, Avadel is continuing the following actions, including those that can potentially accelerate FDA’s final approval decision and shorten the timeline between approval and launch of LUMRYZ:

  • Filed a motion in the U.S. District Court for the District of Delaware on June 23, 2022, to delist the REMS patent from FDA’s Orange Book. A court order requiring the patent holder to delist the REMS patent from the Orange Book could provide a pathway for a final approval of LUMRYZ prior to June 2023.
  • Preparing for a claim construction hearing scheduled for August 31, 2022, that the court previously stated is needed in order to rule on the pending patent delisting motion.
  • Continuing key activities in anticipation of final approval, including planning for the final preparation of the LUMRYZ REMS program and the continued manufacturing of commercial supply.

Avadel estimates that in the last three years, 10,000 to 15,000 patients have discontinued their twice-nightly oxybate use, many due to complications associated with middle of the night dosing. Furthermore, based on an analysis of U.S. claims data, the company believes that each year approximately 3,000 patients initiate oxybate treatment for the first time and expects this to grow by 25% to 50% over time with the introduction of LUMRYZ.

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