FDA News

NeurologyLive recaps September 2026 FDA approvals and their potential implications for the evolving treatment landscape across neurology. [WATCH TIME: 3 minutes]

The FDA set a first quarter 2027 goal date for troriluzole in spinocerebellar ataxia and plans an advisory committee after a 2025 complete response letter.

Fast track status covers AV-1959R, an investigational amyloid-beta vaccine, for preventing Alzheimer disease in cognitively unimpaired adults with amyloid pathology.

Clene has filed an NDA seeking accelerated approval of CNM-Au8 for ALS following 2 missed primary trial end points and years of evolving FDA guidance.

Adults with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive now have a second self-administration option for the complement inhibitor zilucoplan.

Catch up on any of the neurology headlines you may have missed in September 2026, compiled into 1 place by the NeurologyLive® team.

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

FDA approves once-daily oral tavapadon for Parkinson disease, a first-in-class D1/D5 partial agonist that improves motor function and ON time, with key safety warnings.

The FDA extended its review of relutrigine for SCN2A- and SCN8A-related epilepsies by three months, pushing the PDUFA date to December 27, 2026.

Xenon Pharmaceuticals submitted an FDA new drug application for azetukalner, a KV7 potassium channel opener, as adjunctive therapy for focal seizures in epilepsy.

The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.

Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.

The FDA clearance allows AbelZeta to study bispecific CAR T-cell therapy C-CAR168 in refractory progressive multiple sclerosis.

Microstructure Imaging has announced that the FDA cleared its MICSI-PET, an MR-guided PET enhancement platform with automated amyloid Centiloid and tau quantification.

FDA approval of apitegromab introduces a muscle-targeting approach to SMA treatment, with phase 3 data demonstrating motor-function benefit when added to existing SMN2-directed therapies.

The FDA granted priority review to satralizumab for MOGAD, positioning it to become the first approved treatment for the rare autoimmune disease.

Catch up on any of the neurology headlines you may have missed in August 2026, compiled into 1 place by the NeurologyLive® team.

FDA approved Zanvastro (zilganersen), the first therapy to directly target the GFAP protein buildup that drives Alexander disease, based on a phase 3 trial showing improved walking speed and motor function versus untreated controls.

Catch up on any of the neurology headlines you may have missed from last week, compiled into 1 place by the NeurologyLive® team.

Cellenkos’ investigational allogeneic, cord blood-derived T-regulatory cell therapy is being evaluated in a phase 1/1b study in ALS, with early findings showing reductions in plasma neurofilament light chain and increases in IL-10.

The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data.

The FDA has cleared Roche's Elecsys pTau217, a blood test that uses a single biomarker to both rule in and rule out amyloid pathology in adults being evaluated for Alzheimer disease, across both primary and specialty care settings.

FDA has cleared C2N Diagnostics' PrecivityAD2 blood test for adults 40 years and older with cognitive symptoms, offering fast amyloid and tau insight to guide diagnosis and trials in Alzheimer disease.

The FDA granted Fast Track designation to Acadia Pharmaceuticals’ investigational remlifanserin for hallucinations and delusions associated with Alzheimer disease psychosis.

The FDA approved Tauklarify (florquinitau F 18 injection), a tau PET imaging agent, for identifying tau neurofibrillary tangle pathology in adults being evaluated for Alzheimer disease.

The FDA has granted 510(k) clearance to VoxNeuro's Cognitive Function Neuroimaging software, an EEG/ERP-based tool designed to give clinicians an objective, adjunctive measure of cognitive function.

Six FDA PDUFA decisions this fall could reshape care in Alzheimer disease, DMD, Sanfilippo syndrome, Alexander disease, and SMA, here's what clinicians need to know about the data behind each.

The FDA has approved oveporexton, marketed as Orzeyful, as the first therapy to directly restore orexin signaling and address the full symptom range of narcolepsy type 1.