FDA News

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 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.

Biogen plans to resubmit its application for a higher dose of nusinersen after receiving FDA feedback, aiming to enhance SMA treatment efficacy.

Scholar Rock navigates FDA hurdles for apitegromab, a promising treatment for spinal muscular atrophy, aiming for resubmission after addressing manufacturing concerns.

Saol Therapeutics faces challenges after FDA's complete response letter for SL1009, a potential treatment for rare mitochondrial disorder PDCD.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Patricia Pozo-Rosich, MD, PhD. [LISTEN TIME: 19 minutes]

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

Eisai and Biogen seek FDA approval for a convenient, at-home Alzheimer’s treatment option, enhancing patient flexibility and reducing costs.

The autoinjector, branded as Leqembi Iqlik, contains a 360 mg/1.8 mL dose administered over 15 seconds. It is expected to enhance Alzheimer treatment accessibility and offer cost savings for patients.

The decision stemmed from the DREAM study, which found that the Genio system was effective regardless of a patient’s sleeping position, which was particularly important considering patients spend on average 35% to 40% of in a supine position.

PTC Therapeutics faces FDA setback for vatiquinone, a potential Friedreich ataxia treatment, requiring further studies to demonstrate efficacy.

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

Teva's fremanezumab gains FDA approval for pediatric migraine prevention, offering a vital treatment option for children aged 6-17.

The FDA-cleared devices allow for a more simplified administration of the liquid medicine Hyqvia, reducing the number of steps needed to prepare for the infusion of 2 dual vial units or more.

FDA halts Sarepta's Elevidys gene therapy distribution after three patient deaths, prioritizing safety amid ongoing clinical trial evaluations.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Stewart Tepper, MD. [LISTEN TIME: 26 minutes]