John W. Day, M.D. – FDA EIR, Jan 22, 2019

An FDA inspection was conducted at the site of John W. Day, M.D., located in Palo Alto, CA, from January 16 to January 22, 2019. This initial comprehensive inspection, requested by the Center for Biologics Evaluation and Research, focused on Dr. Day's role as a clinical investigator for a Phase 3 gene replacement therapy trial (AVXS-101-CL-303) for spinal muscular atrophy. The study involved an orphan new biologic, with AveXis, Inc. as the sponsor. The inspection reviewed various records, including enrollment, Institutional Review Board (IRB) approvals, subject records, and accountability records, to ensure compliance with clinical investigation regulations. During the review, the investigator found no evidence of adverse events not previously documented and no mismatched data from case files to submitted data. A close-out meeting was held on January 22, 2019, with Dr. Day and Stanford personnel. Importantly, no FDA Form 483 (listing observations of objectionable conditions) was issued, and no verbal items requiring action were discussed, indicating satisfactory adherence to regulatory requirements during the inspection.