FDA News

The 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.

Nerivio, 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.

A 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.

Capsida’s CAP-002 gene therapy, currently advancing through IND-enabling studies, aims to address the unmet needs of patients with STXBP1-related developmental and epileptic encephalopathy by stably supplementing STXBP1 protein.

Catch up on any of the neurology news headlines you may have missed over the course of October 2024, compiled all into one place by the NeurologyLive® team.

Rapport 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.

The NDA includes data from a global placebo-controlled, 72-week study as well as findings from the STRIDE registry, an ongoing, observational, real-world study of ataluren in routine care.

STS101 showed promising results in previous trials, with significant pain relief and minimal adverse events reported, reinforcing its potential as a viable treatment for patients with migraine.

A 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.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Anne Marie Morse, DO, FAASM. [LISTEN TIME: 12 minutes]

Sodium oxybate is the only FDA-approved once-at-bedtime treatment for cataplexy or excessive daytime sleepiness in pediatric patients with narcolepsy.

In 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.

Catch up on any of the neurology news headlines you may have missed over the course of September 2024, compiled all into one place by the NeurologyLive® team.

The decision follows positive data from multiple trials, showing significant improvement in both cardiac function and muscle performance in patients with DMD.

The hope is that NP001 could potentially slow the progression of the disease through its unique immunologic mechanism.

The approval follows phase 3 data that showed significant improvements in neurological symptoms and functional benefit over 12 weeks in patients with Niemann-Pick disease type C.

Zevra 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.

The agent is composed of 2 active ingredients with synergistic anti-oxidant and anti-inflammatory effects which can reduce brain cell injury or impairment caused by acute ischemic stroke.

Approved based on the phase 3 OCARINA II trial, the new subcutaneous formulation offers patients with multiple sclerosis more flexibility to choose treatment options that suit their individual needs.

Catch up on any of the neurology news headlines you may have missed over the course of August 2024, compiled all into one place by the NeurologyLive® team.

The agency’s letter of support for using the α-synuclein seed amplification assay biomarker was supported by data observed in the Parkinson's Progression Markers Initiative cohort study.

Ashwini 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.

Jim 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.

NeuroQuant previously received clearance from the FDA as a medical device intended for automatic labeling, visualization, and volumetric quantification of segmentable brain structures in MRI analysis.

AXS-07 has been supported by data from 2 major phase 3 trials in which treatment with the agent led to significant reliefs in pain freedom, the most bothersome symptoms, and acute medication use.

The 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.

Mirdametinib, 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.

The FDA has granted fast track designation to [18F]PI-2620 for 3 neurodegenerative conditions, highlighting its potential to improve early diagnosis and treatment.

The submission was based on results from the phase 3 Vivacity-MG3 study, which demonstrated nipocalimab’s superiority over placebo when added to standard of care in patients with generalized myasthenia gravis.

Prior to this, the FDA granted regenerative medicine advanced therapy designation to the cell therapy for refractory stiff-person syndrome.