Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
According to a new announcement, the FDA has given clearance to Roche’s Elecsys ptau181 test, marking it the first blood-based biomarker test available for use in primary care settings to assist in Alzheimer disease (AD) assessment and diagnosis. Through this minimally invasive test, clinicians may better identify patients in early stages of cognitive decline who are unlikely to have AD-related amyloid pathology. The Elecsys pTau 181 test, which gained European CE mark earlier this year, is intended for patients aged 55 and older presenting with signs, symptoms, or complaints of cognitive decline. To date, it remains the first and only In Vitro Diagnostic Regulation (IVDR)-certified blood test to help rule out AD-associated amyloid pathology.
Denali Therapeutics announced that the FDA has extended its review period for the company’s biologics license application (BLA) for tividenofusp alfa, an investigational therapy for Hunter syndrome, a rare lysosomal storage disorder. The revised PDUFA date for the therapy, which aims to become the first approved treatment for Hunter syndrome, is now set for April 5, 2026. The agency’s decision to extend the review period comes after Denali submitted additional pharmacology data in response to a routine information request as part of the FDA’s standard evaluation process. Importantly, the agency did not request any new efficacy, safety, or biomarker data as part of this amendment.
Subgroup data presented from the ongoing phase 4 LOTUS trial, a prospective study of individuals with Rett syndrome (RTT) on trofinetide (Daybue; Acadia), revealed that treatment with the medication led to real-world improvements in a number of RTT-related behavioral symptoms among both pediatric and adult patients. Presented at the 2025 Child Neurology Society (CNS) Annual Meeting, held October 8-11 in Charlotte, North Carolina, the subgroup analysis focused on the 12-month follow-up of pediatric (0-17 years of age; n = 117) and adult (≥18 years of age; n = 74) patients within the LOTUS trial. Because of ongoing enrollment, the data reported from the analysis included those who’ve been on trofinetide for 9 months. Here, results showed that treatment with the medication led to improvements in nonverbal communication (pediatric: 53-64%; adult: 41-58%), alertness (pediatric: 50-69%; adult: 33-65%), and social interaction/connectedness (pediatric: 36-58%; adult: 26-48%).
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