FDA News

APN-1607 is undergoing a global phase 3 trial to evaluate its efficacy as an early diagnostic biomarker for progressive supranuclear palsy.

Avidity Biosciences anticipates to provide new cohort data from the phase 1/2 EXPLORE44 trial assessing AOC 1044 in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the second half of 2024.

Labeling updates underscore the importance of using compatible autoinjectors with specific glatiramer acetate injection products to prevent medication errors.

Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.

Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 2024.

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

The FDA AdComm will deliberate over the phase 3 TRAILBLAZER-ALZ 2 trial, a large-scale study that assessed the efficacy and safety of donanemab in patients with early-stage Alzheimer disease over an 18-month period.

Despite failing to meet the primary end point, AMO-02 achieved clinically and statistically significant benefit in various functional and objective assessments in the phase 2/3 REACH-CDM trial.

To date, treatment with foralumab has shown promising results in stabilizing or improving clinical measures without serious adverse effects among patients with non-active secondary progressive multiple sclerosis.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Michael Rogawski, MD, PhD. [LISTEN TIME: 12 minutes]

The original approval for chorea associated with Huntington disease was based on the phase 3 KINECT-HD study, an 128-patient cohort trial in which valbenazine met its primary end point.

The FDA approval of Medtronic’s spinal cord stimulator makes it the first on the market to offer a closed-loop feature that senses biological signals along the spinal cord and automatically adjusts stimulation in real time for chronic pain.

In recent news, Lexeo Therapeutics' gene therapy candidate LX2006 received FDA fast track designation for patients with Friedreich ataxia cardiomyopathy.

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.