FDA News

The approval of donanemab marks the third antiamyloid therapy to gain the FDA's greenlight, following the conditional approval of aducanumab in 2021, and lecanemab in 2023—although aducanumab no longer remains on market after it was removed earlier this year.

In the CRL, the agency cited observations identified during inspection of a third-party manufacturer listed in the NDA. If approved, ABBV-951 would be the first subcutaneous delivery option of carbidopa/levodopa.

Pitolisant is the first and only drug in the class of antagonist/reverse agonists of the histamine H3 receptor for the treatment of patients with narcolepsy.

The therapy is the first and only neonatal Fc receptor blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.

The AAV vector-based gene therapy originally received FDA approval for treating ambulatory pediatric patients aged 4 to 5 years with Duchenne under the accelerated approval pathway.

The cleared phase 1/2 clinical trial will assess SPG302 as a once-a-day pill among patients with amyotrophic lateral sclerosis.

AMT-130, uniQure’s investigational gene therapy, is the first therapeutic candidate to receive regenerative medicine advanced therapy designation for Huntington disease.

Deflazacort, a corticosteroid approved for children with Duchenne muscular dystrophy, has been on the market since 2017 under the name Emflaza.

In a pivotal, large-scale, phase 3 trial, donanemab met its primary end point, demonstrating more pronounced effects in patients with early-stage Alzheimer disease who had low/medium tau status.

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

If the sBLA is approved, the clinical and biomarker benefits may be maintained through the once-monthly dosing regimen that is less burdensome and easier for patients and care partners to continue long-term.

Following presented phase 1 clinical trial findings at AAN 2024, bemdaneprocel receives regenerative medicine advanced therapy designation by the FDA for the treatment of Parkinson disease.

The headset features soft-tip electrodes, artificial intelligence-enabled notifications, and cloud integration for remote monitoring of EEG in clinical practice.

The FDA approval of the increased maximum daily dose for amifampridine offers clinicians and patients greater flexibility in treatment regimens for the management of Lambert-Eaton myasthenic syndrome.

The newly added tablet strengths allow for greater flexibility when selecting or adjusting dosing for deutetrabenazine.

Larimar Therapeutics anticipates interim data from the open label extension study assessing nomlabofusp in patients with Friedreich Ataxia in the fourth quarter of 2024.

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.