FDA News

Alixorexton, an oral investigational selective orexin 2 receptor agonist, is currently in development for the treatment of narcolepsy type 1 and type 2, and idiopathic hypersomnia.

The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.

Stephanie Fradette, PharmD, head of neuromuscular development at Biogen, gave immediate reaction to the EC approval of high-dose nusinersen and its implications for evolving SMA treatment strategies.

The FDA approved Sentynl Therapeutics’ copper histidinate for the treatment of pediatric Menkes disease, a rare genetic neurodegenerative disorder, following an earlier complete response letter.

The FDA is expected to have a decision on efgartigimod as potentially the first approved therapy for seronegative myasthenia gravis by May 10, 2026.

Catch up on any of the neurology news headlines you may have missed in December 2025, compiled into 1 place by the NeurologyLive® team.

The newly approved generic product is expected to perform like other high-quality glatiramer acetate generics, with differences confined mainly to the manufacturer and device, not mechanism or dosing.

Explore anticipated FDA decisions on groundbreaking neurology drugs in 2026, shaping treatment options for various neurologic conditions.

These were the most impactful FDA approval stories that were part of our coverage in 2025, brought to you as part of NeurologyLive's Year in Review.

Inebilizumab is the first CD19-targeted B-cell therapy approved for antiAChR- or antiMuSK antibody–positive generalized myasthenia gravis, providing symptom control with twice-yearly dosing.

The phase 3 study aims to confirm findings from the HEALEY ALS platform trial in patients with early-stage rapidly progressive ALS, which suggested potential benefits of pridopidine across multiple domains.

Catch up on any of the neurology news headlines you may have missed in November 2025, compiled into 1 place by the NeurologyLive® team.

TRN-257, a treatment in development for adults with narcolepsy and idiopathic hypersomnia, is designed to reduce daily sodium exposure relative to existing higher-sodium therapies.

uniQure navigates FDA's unexpected shift on AMT-130, aiming for accelerated approval for Huntington's disease despite recent regulatory challenges.

The FDA has approved Novartis’ intrathecal formulation of onasemnogene abeparvovec-brve, marketed as Itvisma, for patients ages 2 years and older living with spinal muscular atrophy.

The agency has required a postmarketing observational study to monitor safety for at least 12 months following gene therapy treatment in patients with Duchenne muscular dystrophy.

Jupiter Neurosciences advances Parkinson treatment with FDA clearance for JOTROL's phase 2a trial, showcasing promising safety and bioavailability results.

Catch up on any of the neurology news headlines you may have missed in October 2025, compiled into 1 place by the NeurologyLive® team.

Axsome Therapeutics advances AXS-05 for Alzheimer agitation, showcasing promising trial results and potential for treating major depressive disorder.

Feedback from the FDA allows CervoMed to move forward with the proposed primary and secondary end point of the phase 3 trial assessing neflamapimod in dementia with Lewy bodies.

FDA issues a complete response letter for Biohaven's troriluzole, a potential treatment for spinocerebellar ataxia, citing data concerns.

UCB's Kygevvi becomes the first FDA-approved treatment for thymidine kinase 2 deficiency, offering hope to patients with this rare neuromuscular disorder.

The FDA accepts Lantheus' MK-6240 application, a promising PET imaging agent for Alzheimer disease, enhancing diagnosis and treatment strategies.

The FDA has granted fast track designation to Eisai’s etalanetug, an anti-tau antibody targeting tau pathology in Alzheimer's disease.

Roche's Elecsys pTau181 test receives FDA clearance, revolutionizing Alzheimer diagnosis in primary care with a minimally invasive blood test.

Denali Therapeutics prepares for potential FDA approval of tividenofusp alfa, a groundbreaking therapy for Hunter syndrome, with a revised review date set.

Catch up on any of the neurology news headlines you may have missed in September 2025, compiled into 1 place by the NeurologyLive® team.

AbbVie’s application to the FDA for its selective dopamine D1/D5 receptor partial agonist is supported by data from the phase 3 TEMPO study program.