August was a landmark month for FDA neurology actions. The agency approved oveporexton (Orzeyful, Takeda) for narcolepsy type 1 in adults — the first drug to directly restore orexin signaling, the core biological deficit in the disorder. The twice-daily tablet, which earned Breakthrough Therapy designation and Priority Review, improved daytime sleepiness, cataplexy, sleep paralysis, hallucinations, and disrupted sleep in two clinical trials.

On the Alzheimer's front, the FDA cleared two new blood tests: the Elecsys Phospho-Tau (217P) Plasma test (Roche/Eli Lilly) for adults 55+ with cognitive decline — the first cleared for both rule-in and rule-out assessment using the same cutoffs across care settings — and PrecivityAD2 (C2N Diagnostics), the only test available for adults as young as 40. The FDA also approved Tauklarify, a tau-targeted PET imaging agent for evaluating AD in adults with cognitive impairment.

Key Takeaways:

• Oveporexton is the first therapy to target orexin signaling directly, offering a mechanistically novel option for narcolepsy type 1
• Two cleared Alzheimer's blood tests expand diagnostic access across age groups and care settings
• A clinical hold was placed on RGX-121, a gene therapy for Hunter syndrome, after asymptomatic spinal MRI abnormalities were found in 5 trial participants
• CK0803, a regulatory T-cell therapy for ALS, received FDA Fast Track designation — the first of its kind for ALS

Why it matters: These actions collectively expand diagnostic and therapeutic options for some of neurology's most challenging conditions, while the Hunter syndrome hold is a reminder of the safety vigilance required in gene therapy development.