Regulators at the Food and Drug Administration have approved a new blood test for Alzheimer’s disease, while also extending deadlines for several other drug candidates and halting a gene therapy trial. The agency cleared Elecsys pTau217, a blood-based immunoassay developed by Roche, to help clinicians identify amyloid pathology in adults 55 and older showing signs of cognitive decline. The test is described as the first and only FDA-cleared, single-biomarker blood test that supports both rule-in and rule-out assessments using the same validated cutoffs across primary and specialty care settings.
The FDA also took action on several other neurology-related products this week. The agency granted Fast Track Designation to safusidenib, an investigational oral inhibitor of mutant IDH1, for the treatment of IDH1-mutant glioma. The designation follows the agent’s advancement into a key phase 3 program. In a separate decision, the administration extended the PDUFA target action date for Capricor Therapeutics’ deramiocel, an investigational cell therapy for Duchenne muscular dystrophy, pushing the deadline from August 22 to November 22, 2026. The delay was attributed to the acceptance of additional phase 3 data.
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Conversely, the FDA placed a clinical hold on clemidsogenelanparvovec, or RGX-121, an investigational gene therapy for Hunter syndrome, after spinal MRI abnormalities were detected in five participants from the CAMPSIITE study. REGENXBIO stated it does not expect to resubmit a biologics license application for the therapy in the near term.
A coalition of Charcot-Marie-Tooth disease organizations, clinicians, and pharmaceutical companies has published the first consensus clinical trial framework for the condition. The framework, developed by ToPIC: CMT, is intended to give drug developers a clearer roadmap as they design trials and pursue FDA review for a disease that currently has no approved treatments. The guidance recommends more flexible trial designs to limit placebo exposure, broader enrollment based on clinical phenotype, and the use of disease-specific, function-based endpoints. It also highlights the potential role of biomarkers such as neurofilament light chain and MRI-based muscle fat fraction in supporting dose selection and prognosis.
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For trainees entering brain injury medicine, the field offers opportunities across inpatient and outpatient care, but the evolving setting can present challenges. Erika Trovato, DO, MS, a brain injury specialist and program director of the Brain Injury Medicine Fellowship at Burke Rehabilitation Hospital, emphasized the importance of remaining curious and open-minded while building expertise and professional relationships. She advised early-career physicians to pace themselves and seek mentorship to handle the expanding scope of the specialty.
The success of the CMT framework will depend heavily on how consistently the industry adopts its recommendations for flexible enrollment and function-based endpoints.