FDA EIR - Michael J. Polydefkis, M.D. - August 20, 2021

An FDA inspection of Clinical Investigator Michael J. Polydefkis, M.D., in Baltimore, MD, was conducted from August 16 to August 20, 2021. The inspection assessed the site's execution of the ALN-TTRSC01-002 (HELIOS-A) clinical study, involving the investigational drug vutrisiran, guided by the Clinical Investigators Compliance Program. The regulatory framework for this inspection was related to an Investigational New Drug application.

While no formal FDA-483 observations were issued, verbal observations were discussed with Dr. Polydefkis. The main issues identified related to incomplete or missing source documentation for efficacy endpoints, specifically Quantitative Sensory Testing (QST) results for one subject and Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) assessments for another. Additionally, inaccuracies were noted on FDA Form 1572 regarding the listing of sub-investigators. A staff role change for a research coordinator, from technician duties to solely office duties, was also discussed to ensure protocol compliance.

Dr. Polydefkis acknowledged these observations. As no formal warnings were issued, the required actions are implicitly to improve adherence to documentation standards for all study records and ensure accurate completion of regulatory forms, thereby reinforcing compliance with clinical trial protocols and applicable regulations.