Avadel Cuts Costs to Pursue U.S. Marketing Approval of Narcolepsy Candidate

Irish biotech Avadel Pharmaceuticals said it is taking steps, including reducing its workforce by 50 percent, to explore every available pathway to accelerate a decision by the U.S. Food and Drug Administration to grant final approval of its lead drug candidate FT218 for the treatment of narcolepsy prior to June 2023.

Photo: Greg Divis, CEO of Avadel Pharmaceuticals

Concurrent with this strategy, Avadel has received and agreed upon what is expected to be a final label and is completing the last edits of the Risk Evaluation and Mitigation Strategy (REMS) with FDA and expects to receive tentative approval of FT218.

“We believe the receipt of a tentative approval for the FT218 New Drug Application (NDA) will validate the clinical efficacy and safety profile of FT218 for people living with narcolepsy and will provide clarity on the timing and pathway to a potential final approval and subsequent commercial launch,” said Greg Divis, CEO of Avadel Pharmaceuticals. “As part of these efforts, we are optimizing our cost structure by focusing our existing resources on our most important priorities, thus extending our cash runway and subsequent financial bridge to a potential final approval in June 2023 or sooner.”

Narcolepsy is a neurological sleep disorder characterized by chronic, excessive attacks of drowsiness during the day, sometimes called excessive daytime sleepiness (EDS). Attacks of drowsiness may persist for only a few seconds or several minutes. These episodes vary in frequency from a few incidents to several during a single day. Nighttime (nocturnal) sleep patterns may also be disrupted. Three additional symptoms often associated with narcolepsy are sudden extreme muscle weakness (cataplexy), a specific type of hallucination that occurs just before falling asleep or upon awakening, and brief episodes of paralysis while waking up. The incidence of narcolepsy is approximately 1 in 2,000.

FT218 is an investigational formulation of sodium oxybate leveraging the company’s proprietary drug delivery technology and designed to be taken once at bedtime for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy.

In March 2020, Avadel completed the REST-ON trial, a randomized, double-blind, placebo-controlled, pivotal phase 3 trial, to assess the efficacy and safety of FT218 in adults with narcolepsy. Among the three co-primary endpoints, FT218 demonstrated statistically significant and clinically meaningful results in EDS, the clinician’s overall assessment of the patient’s functioning, and reduction in cataplexy attacks for all three evaluated doses compared to placebo. Avadel is currently evaluating the long-term safety and tolerability of FT218 in the open-label RESTORE clinical study.

In January 2018, the FDA granted FT218 Orphan Drug designation for the treatment of narcolepsy based on the plausible hypothesis that FT218 may be clinically superior to the twice-nightly formulation of sodium oxybate already approved by the FDA for those with narcolepsy due to the consequences of middle-of-the-night dosing of the approved product. The NDA for FT218 is currently under review by the FDA.

Author: Rare Daily Staff