FDA News

The FDA has approved a label update on the administration options for SK Life Science’s cenobamate, a treatment approved for adults with partial-onset seizures.

The new double-blind, placebo-controlled study includes approximately 200 patients with ALS who will undergo a single bone marrow aspiration procedure to procure the mesenchymal stem cells that will be used to manufacture each participant’s NurOwn treatment for the trial.

The executive vice president and chief research officer of the Muscular Dystrophy Association shared her reaction to the recent FDA approval of givinostat for Duchenne muscular dystrophy.

The first patient enrolled into the phase 1/2a ArthemiR trial assessing ATX-01 myotonic dystrophy type 1 is expected in the second quarter of 2024.

The newly cleared i-STAT TBI test can be used to help assess patients up to 24 hours after injury at patient bedside, with lab-quality results produced in 15 minutes.

SPN-830, an apomorphine infusion device, had its new drug application submitted using data from the phase 3 TOLEDO trial.

The phase 1 study, dubbed PRO-101, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both patients with ALS and healthy volunteers.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Sharon Hesterlee, PhD. [LISTEN TIME: 10 minutes]

The approval is supported by phase 3 data which showed ravulizumab-cwvz met its primary end point of time to first on-trial relapse, with no relapses observed in 58 patients with NMOSD over a median treatment duration of 73 weeks.

Branded as Duvyzat, givinostat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

Revance’s daxibotulinumtoxinA-Ianm (Daxxify) is the first and only approved peptide-formulated, long-lasting neuromodulator for patients with cervical dystonia.

The FDA recently issued revised draft guidance to assist drug companies in developing treatments for early Alzheimer disease, focusing on diagnostic criteria, clinical staging, and outcome measures.

The delivery device is prefilled with the prescribed dose of Acthar Gel in 40- or 80-unit versions, and may help administration of the appropriate subcutaneous dose of Acthar Gel.

In recent news, the FDA has issued a complete response letter for Vanda Pharmaceuticals' tasimelteon for treating insomnia because of identified deficiencies and cannot be approved in its current form.

GA Depot, composed of extended-release microspheres administered intramuscularly, typically every 28 days, was expected to result in fewer injection site reactions than other glatiramer acetate products.

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

The antisense oligonucleotide will continue to be assessed in a large-scale phase 3 study, CARDIO-TTRansform, which will have data read out in 2025.

The FDA has accepted Sarepta Therapeutics' efficacy supplement for gene therapy SRP-9001, granting it a priority review for the treatment of Duchenne muscular dystrophy, with a decision expected by June 21, 2024.

The chief executive officer of ClearPoint Neuro provided clarity on how the recently approved SmartFrame system can enhance the efficiency of operating room procedures, allowing for flexible workflows and image-based corrections.

Encoded Therapeutics plans to initiate clinical trials assessing its gene therapy candidate ETX101 for the treatment of SCN1A+ Dravet syndrome during the first half of 2024.

VCA-894A, a novel antisense oligonucleotide, exhibits a mechanism of action that specifically targets a cryptic splice site variant within immunoglobulin mu-binding protein 2.

Take a look at some of the most-anticipated FDA pending approvals expected in 2024 that researchers and clinicians in neurology should keep an eye out on.

Takeda's FDA-approved Gammagard Liquid can be used as induction therapy, which includes an induction dose followed by maintenance doses, for adults with chronic inflammatory demyelinating polyneuropathy.

The dihydroergotamine product’s application was supported by phase 1 comparative pharmacokinetic and safety data, and the phase 3 ASCEND trial.

Aruna Bio's investigational treatment, AB126, is now set for a phase 1b/2a clinical trial targeting acute ischemic stroke, anticipated to begin in the first half of 2024.

Takeda's Hyqvia is the only FDA-approved combination of immunoglobulin and hyaluronidase, which makes it a facilitated subcutaneous immunoglobulin infusion, for patients with chronic inflammatory demyelinating polyneuropathy.

The newly FDA-approved AI-powered software platform BrainSee utilizes brain MRI and cognitive assessments to reportedly predict the progression of amnestic mild cognitive impairment to Alzheimer disease in 5 years.

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

Months after the FDA cleared a phase 2 study in myasthenia gravis, KYV-101 is set to be assessed in a diverse cohort of refractory progressive multiple sclerosis.