FDA News

The associate professor in pain management and emergency medicine at UC San Diego shared her reaction to the FDA approval of suzetrigine, a selective, nonaddictive alternative to opioids for acute pain management. [WATCH TIME: 4 minutes]

Entrada Therapeutics has recently received FDA clearance to move forward with a clinical trial for its investigational Duchenne muscular dystrophy therapy.

The approval was based on data from the ADAPT-PD trial, a prospective study that laid the foundation for aDBS use in a real-world clinical environment.

The professor of neurology at the Geisel School of Medicine at Dartmouth talked about how the combination therapy involving meloxicam and rizatriptan may provide a more effective approach to treating migraine. [WATCH TIME: 6 minutes]

Marketed as Wakix, the treatment is currently approved in the United States for excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy and for EDS in pediatric patients aged 6 and older.

The director of the Montefiore Headache Center at Albert Einstein College of Medicine provided an overview of the drug profile for the recently approved migraine treatment AXS-07. [WATCH TIME: 3 minutes]

The chair of neurosurgery at the University of Arizona talked about the newly approved infusion therapy that offers continuous medication delivery to improve both motor and non-motor symptoms in Parkinson disease. [WATCH TIME: 5 minutes]

Julie Pilitsis, MD, PhD, MBA, chair of neurosurgery at The University of Arizona Tucson, discussed the impact of a newly approved continuous infusion therapy for Parkinson disease.

Jonathan Rubin, MD, MBA, chief medical officer at Supernus Pharmaceuticals, talked about the recent approval of the company’s subcutaneous infusion therapy SPN-830 for Parkinson disease.

Catch up on any of the neurology news headlines you may have missed over the course of January 2025, compiled all into one place by the NeurologyLive® team.

The director of the Montefiore Headache Center at Albert Einstein College of Medicine discussed the recent FDA approval of a new migraine treatment that combines rizatriptan and meloxicam. [WATCH TIME: 5 minutes]

SPN-830 is a wearable subcutaneous infusion device designed to deliver continuous treatment throughout the waking day, ensuring more consistent control of OFF time.

Suzetrigine, a selective inhibitor of the NaV1.8 sodium channel, offers an alternative opioid treatment option for patients to treat moderate to severe pain without risk of addiction.

Findings from previous phase 3 trial showed that treatment with AXS-07 resulted in significant reductions in headache pain freedom, migraine symptoms, and rescue medication use.

The company announced the completion of its phase 2 study assessing SPG601 in adult men with FXS, with topline findings expected to be reported by end of the first quarter in 2025.

Results from the open-label phase 1/2 study of tividenofusp alfa demonstrated positive effects on evidence-based surrogate end points in participants with Hunter syndrome.

The Delphi-MD platform is designed for nerve stimulation in diagnostic applications, offering clinicians a tool to support objective and personalized treatment approaches in brain health.

Results from Vigil Neuroscience’s ongoing phase 1 trial of VG-3927 in healthy volunteers supported the continued development of the therapy as a potential once-daily oral treatment for Alzheimer disease.

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

These were the most-read FDA approval stories that were part of our coverage in 2024, brought to you as part of NeurologyLive®'s Year in Review.

Adults with obstructive sleep apnea who receivied tirzepatide achieved an average of up to 20% weight loss and experienced at least 25 fewer breathing interruptions for each hour slept.

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

As of July 2024, 2 participants in the ongoing CONNECT1 study have received 4 doses of PGN-EDO51 at 10 mg/kg, which has been generally well tolerated, with initial results expected in early 2025.

Most recently reported topline data from the RewinD-LB phase 2b trial in patients with dementia with Lewy bodies showed that neflamapimod failed to meet its primary end point and secondary end points.

Findings from the ongoing PERSEUS phase 3 study of tolebrutinib in primary progressive multiple sclerosis are anticipated to be reported in the second half of 2025.

The FDA recommended a follow-up meeting with Clene to further discuss the analyses needed to support the accelerated approval pathway for CNM-Au8 in amyotrophic lateral sclerosis.

The FDA has agreed that a scale used in ongoing phase 1/2 clinical trials could potentially serve as an intermediate clinical end point for accelerated approval pathway for uniQure’s investigational gene therapy AMT-130.

A 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.