FDA EIR - Sarepta Therapeutics, Inc. - April 12, 2018

An FDA inspection of Sarepta Therapeutics, Inc. was conducted from April 9-12, 2018, focusing on Postmarketing Adverse Drug Experience (PADE) reporting requirements under FDA Compliance Program 7353.001. This routine inspection, the company's first by the FDA, covered procedures, case files, training, annual reports, and safety signaling related to their sole approved product, Exondys 51. The primary observation noted on Form FDA 483 was Sarepta's failure to ensure proper submission of at least 30 non-expedited Individual Case Safety Reports (ICSRs) within periodic reports. Although these ICSRs were sent via the Electronic Submission Gateway, the company could not provide FDA Adverse Event Reporting System (FAERS) acknowledgments, indicating these reports were not successfully processed into the FDA's database, a violation of 21 CFR 314.80(c)(2)(ii)(B). Additionally, management was advised that Sarepta’s standard operating procedures for collecting follow-up information on adverse events lacked specific timelines for queries. In response, Sarepta management committed to submitting a written reply to the FDA within 15 business days. They plan to investigate the root cause for the missing FAERS entries, involving their external contractors, resubmit affected ICSRs, and integrate a timeline for information queries into their Adverse Event Management Plan and SOPs. These actions are part of a preliminary corrective and preventive action (CAPA) plan.