FDA EIR - Sarepta Therapeutics, Inc. - April 18, 2019

An FDA inspection of Sarepta Therapeutics, Inc. in Cambridge, MA, was conducted from April 15-18, 2019. This inspection evaluated the company's functions as both a Bioanalytical Bioequivalence facility and a clinical study Sponsor, under the Bioresearch Monitoring Compliance Program. Although no Form FDA 483 was issued, several key observations were discussed with management. In the bioanalytical assessment, it was noted that alpha actinin loading control bands were saturated during western blot analysis of dystrophin, impairing accurate protein level evaluation. Additionally, the densitometry software lacked metadata capture and its security module was not utilized for audit trails. Regarding sponsor responsibilities, the inspection found that subjects were underdosed at clinical sites due to pharmacists incorrectly applying dosage rounding rules. These underdosing instances were not consistently reported as major protocol deviations in interim study reports. An unblinding incident at site 401 also required accurate reporting. Sarepta's management acknowledged these findings, committing to ensure appropriate reporting of underdosing and the unblinding incident in the final Case Study Report, and to enhance analytical procedures and data security for upcoming studies.