The FDA had a busy November 2024, making key decisions on potential new therapeutic agents. Approvals, clearances, and a complete response letter were issued, impacting the landscape of neurology and beyond. Here's a roundup of the key actions.
AI Tool Cleared for Safer ARIA Detection in Alzheimer's Treatment
The FDA cleared icobrain aria (icometrix), the first artificial intelligence (AI) software designed to detect, measure, and grade amyloid-related imaging abnormalities (ARIA) associated with new anti-amyloid treatments for Alzheimer's disease (AD). Trained using thousands of brain MRI scans, the software generates a quantitative summary report integrated into hospital workflows. For ARIA-edema (ARIA-E), the software measures the number of ARIA-E sites, their locations, and the longest axis of the largest site, combining these into an area severity score graded according to clinical guidelines. For ARIA-hemorrhages (ARIA-H), the software counts and localizes new microhemorrhages and areas of superficial siderosis, reporting an ARIA-H severity score graded per clinical guidelines. Stephen Salloway, MD, professor of neurology and psychiatry at the Warren Alpert Medical School of Brown University, noted that new standardized tools like icobrain aria are needed to assist radiologists and clinicians in detecting and managing ARIA to optimize patient safety.
Nerivio Receives Expanded Age Indication for Migraine Treatment
Theranica's Nerivio remote electrical neuromodulation (REN) wearable received an expanded age indication from the FDA, now approved for patients aged 8 years and older with acute migraine. This makes it the first cleared non-drug therapy for acute migraine treatment in pediatric patients and the only prescribed preventive migraine treatment in this age group. The decision was supported by a study (NCT06138756) led by Klaus Werner, MD, PhD, a pediatric neurologist at Duke Health, which demonstrated the safety and efficacy of the REN device for treating acute migraine in pediatric patients aged 6 to 11 years. The study, published in the Annals of the Child Neurology Society, included 293 patients with a median age of 11 years. 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. Werner highlighted the uniqueness of Nerivio as a novel treatment for migraine, especially for children 8 years of age and above, now approved for both acute and preventative treatment.
Accelerated Approval for Gene Therapy in AADC Deficiency
PTC Therapeutics' gene therapy eladocagene exuparvovec-tneq received accelerated approval from the FDA for treating patients aged 18 years and older with aromatic L-amino acid decarboxylase (AADC) deficiency, including the full spectrum of disease severity. Marketed under the name Kebilidi, this marks the first gene therapy directly administered to the brain approved in the United States. AADC deficiency is a rare, fatal genetic disorder causing severe disability and suffering from the first months of life, leading to decreased muscle tone, movement disorders, and disruption of the autonomic nervous system. Matthew B. Klein, MD, chief executive officer at PTC Therapeutics, expressed pride in the team's commitment to achieving this regulatory milestone and looked forward to bringing this transformational gene therapy to children and adults with AADC deficiency in the United States.
