Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
The FDA has approved safety-related labeling updates for delandistrogene moxeparvovec-rokl (Elevidys; Sarepta Therapeutics), a marketed gene therapy for Duchenne muscular dystrophy (DMD), to include a new boxed warning and a restriction limiting its treatment use for ambulatory patients ages 4 and older with the condition. The decision stems after recently reported cases of fatal acute liver failure for patients on the treatment. The determination is a result of a CBER (Center for Biologics Evaluation and Research) Safety Communication issued by the FDA in June, which reported 2 fatal cases of acute liver failure in nonambulatory pediatric boys treated with the gene therapy. Responding to these events, Sarepta voluntarily paused the distribution of the gene therapy for nonambulatory patients.
Using registry data from the Piemonte and Valle d'Aosta region of Italy (PARALS), a newly published study in Annals of Clinical and Translational Neurology reported that the global prevalence of amyotrophic lateral sclerosis (ALS) is projected to increase significantly by 2040, mainly driven by incidence associated with population aging and gradual improvements in survival. Overall, these findings underscore the potential need for public health policies to promptly allocate resources to ensure the necessary care for patients with ALS. In this study, researchers explored trends in incidence, survival, and prevalence of ALS between 2005 and 2019 from the PARALS registry (n = 3294) and then estimated these trends for future years up to 2040. Based on the observed projections, findings suggest that the prevalence of ALS is expected to increase in other countries, with a median rise of 24.9% from 2024 to 2040 and 55.6% from the last available estimates to 2040; however, the authors noted that the impact might differ based on the size of the population.
According to a recent company update, Clene Nanomedicine plans to submit a new drug application (NDA) in the first quarter of 2026 for its investigational, first-in-class, nanocatalytic, bioenergetic agent CNM-Au8 as a treatment for amyotrophic lateral sclerosis (ALS). The company is planning to request an additional type C meeting with the FDA to discuss data for the submission, which is expected to be through the accelerated approval pathway. CNM-Au8 is an oral suspension of clean-surfaced faceted gold nanocrystals designed as a bioenergetic/neuroprotective therapy for neurodegenerative diseases. The agent aims to restore energetic homeostasis and reduce oxidative stress in neurons and glia, with preclinical models showing remyelination and neuroprotection in demyelinating and ALS models. Within its ALS program, the drug has been tested in the phase 2 RESCUE-ALS trial, HEALEY-ALS platform study, and through an expanded access program.
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