The NeurologyLive FDA News page houses all of our coverage of FDA-related actions, from new drug approvals and advisory committee meetings to application submissions, clinical holds, trial clearances, and beyond.
FDA Grants NS-050/NCNP-03 Rare Pediatric Disease Designation in Duchenne Muscular Dystrophy
December 5th 2024A recently initiated phase 1/2 trial of NS-050/NCNP-03 will assess patients with Duchenne muscular dystrophy on dystrophin production, muscle strength, mobility, and functional exercise capacity.
FDA Clears Icobrain Aria, First AI Tool for Safer ARIA Detection in Alzheimer Treatment
November 14th 2024The software, designed using thousands of brain MRI scans, is used for automated detection and severity grading of ARIA-E and ARIA-H for safety monitoring of new Alzheimer disease therapies.
FDA Approves Expanded Age Indication for Nerivio Migraine Device in Pediatric Patients
November 14th 2024Nerivio, a nondrug option for acute migraine attacks and prevention of migraine with or without aura, has been FDA-approved for several years in adolescents aged 12 and older as well as adults.
FDA Grants Accelerated Approval to PTC Therapeutics’ Gene Therapy for AADC Deficiency
November 13th 2024A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.
FDA Places Clinical Hold on Epilepsy Agent RAP-219 for Diabetic Peripheral Neuropathic Pain
November 7th 2024Rapport Therapeutics’ is actively recruiting patients for its phase 2a trial investigating its lead therapy candidate RAP-219 among patients with focal epilepsy, with topline data anticipated in mid-2025.
Eisai Formally Submits BLA for Subcutaneous Autoinjector of Lecanemab
November 1st 2024A subgroup analysis of the phase 3 Clarity AD open-label extension study showed a 14% increased amyloid plaque removal with subcutaneous lecanemab vs intravenous administration, the administration for which it was FDA-approved for.
FDA Approves AbbVie’s 24-Hour Foscarbidopa/Foslevodopa Pump for Advanced Parkinson Disease Treatment
Published: October 17th 2024 | Updated: October 21st 2024In clinical trials, patients with Parkinson disease who were treated with foscarbidopa/foslevodopa experienced superior improvement in ON time without dyskinesia compared with oral immediate-release carbidopa/levodopa.
FDA Approves Zevra Therapeutics’ Arimoclomol as First Treatment for Niemann-Pick Disease Type C
September 25th 2024Zevra Therapeutics plans to immediately start its launch activities for arimoclomol, marketed as Miplyffa, which is anticipated to be commercially available in 8 to 12 weeks in the United States.
Significance of Medtronic’s DBS System Approved for Asleep Neurosurgery
September 8th 2024Ashwini Sharan, MD, chief medical officer of Medtronic Neuromodulation, provided commentary on the FDA’s decision to approve asleep deep brain stimulation for the treatment of Parkinson disease and essential tremor.
Potential of Advancing Neurofibromatosis Care With Investigational Mirdametinib
September 4th 2024Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, talked about how the prioritization of mirdametinib, a promising treatment for both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.
FDA Clears Clearmind Biomedical’s Neuroblade System for Minimally Invasive Neurosurgery
September 3rd 2024The system is composed of the Neuroblade, a single-use multifunctional neuroendoscope; the Neuropad, a reusable medical-grade tablet; and Clearpath, a disposable transparent access sheath.
FDA Grants Priority Review to New Drug Application of Neurofibromatosis Agent Mirdametinib
August 31st 2024Mirdametinib, a treatment in development for adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas, was granted a PDUFA target action date of February 28, 2025.