FDA Approves Lumyrz for Pediatric Narcolepsy, 24-Hour Foslevodopa/Foscarbidopa Gets FDA Greenlight, Oral Edaravone Fails in Phase 3 Study

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio.

According to a new announcement, the FDA has approved Avadel Pharamaceuticals' supplemental new drug application (sNDA) for sodium oxybate (Lumryz) for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. With this approval, sodium oxybate could alleviate the burden placed on families and caregivers of pediatric patients with narcolepsy who are responsible for waking up in the middle of the night to administer a second dose. Lumryz comes with a boxed warning as a central nervous system depressant, and for its potential abuse and misuse. It is currently only available through a restricted program under a Risk Evaluation and Mitigation Strategy called the Lumryz REMS. With the approval, the drug was granted 7 years of orphan drug exclusivity.

According to a new announcement, the FDA has approved AbbVie’s foscarbidopa/foslevodopa (Vyalev), also known as ABBV-951, as the first and only subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations in adults with advanced Parkinson disease (PD). The company noted that timing for a patient's access to the therapy is dependent on their individual insurance plan, with coverage for Medicare patients expected in the second half of 2025.The approval was supported by data from the phase 3 M15-736 study (NCT04380142) that assessed the efficacy of continuous subcutaneous infusion of foscarbidopa/foslevodopa among adult patients with advanced PD compared with oral immediate-release carbidopa/levodopa (CD/LD IR), along with a 52-week, open-label M15-741 study (NCT03781167) which investigated the long-term safety and efficacy of the treatment. Results from the M15-736 study revealed that patients who received foscarbidopa/foslevodopa reported superior improvement in motor fluctuations, with increased ON time without troublesome dyskinesia and decreased OFF time, compared with oral CD/LD IR.

New findings from the phase 3b study MT-1186-A02 (NCT04569084) assessing an investigational daily dosing of oral edaravone (Radicava ORS; Mitsubishi Tanabe Pharma America) showed that this form of the treatment was not superior in efficacy at 48 weeks compared with the on/off regimen of edaravone in patients with amyotrophic lateral sclerosis (ALS). Presented at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) meeting, held October 15-18, in Savannah, Georgia, these findings reinforce the appropriateness of the FDA-approved regimen.In the analysis, results revealed that daily dosing did not show achieve a statistically significant difference versus on/off dosing with oral edaravone at week 48 on the combined assessment of function and survival (CAFS), which included change in ALS Functional Rating Scale-Revised (ALSFRS-R) and time to death.

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