Agios Pharmaceuticals, INC. – FDA EIR, Sep 16, 2021

An FDA PDUFA surveillance inspection of Agios Pharmaceuticals, Inc. was conducted from September 7-16, 2021, focusing on the company's conduct of two clinical study protocols (AG348-C-006 and AG348-C-007). The inspection, part of the BIMO FY 21 workplan under compliance program 7348.810 for Sponsors, Contract Research Organizations, and Monitors, identified two significant discussion items, although no Form FDA 483 was issued. The primary issues involved the sponsor's and its Contract Research Organization's failure to ensure timely compliance at a specific clinical site (840104). This included persistent problems with the principal investigator's unavailability during monitoring visits, delayed review of laboratory reports, and a failure to review Investigational New Drug (IND) safety reports, some of which were Suspected Unexpected Serious Adverse Reactions (SUSARs). Additionally, it was noted that individuals not identified as members of the Independent Data Monitoring Committee (IDMC) attended closed-door meetings where unblinded study data was presented and discussed, raising concerns about data integrity and blinding protocols. Agios Pharmaceuticals acknowledged these discussion items and provided an immediate written response during the inspection, but declined to submit further post-inspection documentation. The FDA investigator emphasized the potential for future regulatory classifications, including Official Action Indicated (OAI), which could lead to warning letters or untitled letters, should these issues not be adequately addressed.