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Getting the latest healthcare news for you
Getting the latest healthcare news for you
The FDA had a busy August in neurology. A first-in-class narcolepsy drug that restores orexin signaling was approved, two new Alzheimer's blood tests received clearance (one for adults as young as 40), and a tau-targeting PET imaging agent got the green light — while a gene therapy for Hunter syndrome hit a clinical hold.
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:
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.
