FDA Clears ARIA Detecting Software, Nerivio Gains Expanded Indication, FDA Approves First-Ever Gene Therapy for AADC Deficiency

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

According to an announcement, the FDA has cleared icobrain aria (icometrix), the first artificial-intelligence (AI) software approved to detect, measure, and grade amyloid-related imaging abnormalities (ARIA), a potential serious adverse effect of new anti-amyloid treatments for Alzheimer disease (AD).This software, trained using thousands of brain MRI scans, generates a quantitative summary report with includes a severity rating score that is integrated into the hospital workflow. For ARIA-edema (ARIA-E), the number of ARIA-E sites, their locations, and the longest axis of the largest site are measured and combined into an area severity score, graded according to clinical guidelines. For ARIA-hemorrhages (ARIA-H), the number of new microhemorrhages and areas of superficial siderosis are individually counted and localized, with an ARIA-H severity score reported, graded per clinical guidelines.

According to a new announcement, the FDA has approved an expanded age indication for Theranica's Nerivio remote electrical neuromodulation (REN) wearable to treat patients aged 8 years and older with acute migraine. With this expanded indication, the needle-free alternative treatment becomes the first cleared nondrug therapy for acute migraine treatment in pediatric patients and the only prescribed preventive migraine treatment in this patient age group.This decision comes following a recently published study (NCT06138756) led by Klaus Werner, MD, PhD, a pediatric neurologist at Duke Health, which showed that treatment with the REN device was safe and effective for treating acute migraine in pediatric patients aged between 6 years and 11 years. Published in the Annals of the Child Neurology Society, the study featured 293 patients with a median age of 11 years who used the REN device to treat at least 1 migraine attack. Of them, 18.4% reported at least 1 attack with aura, and the remaining 81.6% never reported aura. Notably, two-thirds (67.4%) of REN treatments were not followed by another REN treatment in 24 hours, indicating no retreatment and implying no recurrence in 24 hours.

According to a new announcement, the FDA has granted PTC Therapeutics’ gene therapy eladocagene exuparvovec-tneq an accelerated approval indicated for the treatment of patients aged 18 years and older with AADC deficiency, including the full spectrum of disease severity. Marketed under Kebilidi, it becomes the first-ever gene therapy that is directly administered to the brain approved in the United States.AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, leading to decreased muscle tone, movement disorders, and disruption of the autonomic nervous system. The company reported that launch preparations are progressing smoothly, with centers of excellence designated and surgeons already trained to administer the gene therapy.

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