FDA EIR - Mirum Pharmaceuticals, Inc. - May 12, 2021

Mirum Pharmaceuticals, Inc. underwent an FDA Bioresearch Monitoring (BIMO) sponsor inspection from May 5 to May 12, 2021, at its Foster City, CA facility. This comprehensive review, conducted under Compliance Program 7348.810, evaluated Mirum's responsibilities as a sponsor for its investigational product Maralixibat (IND 119917, NDA 214662), intended for Alagille Syndrome, under the Food, Drug, and Cosmetic Act.

The inspection focused on areas such as investigator and monitor selection, clinical trial monitoring, ClinicalTrials.gov registration, quality assurance, safety and adverse event reporting, financial disclosure, and investigational product accountability. Importantly, the inspection concluded without the issuance of a Form FDA 483, Inspectional Observations, indicating that no significant regulatory violations were identified by the FDA during this review. No specific discussion items or refusals were encountered, and no samples were collected.

The firm had proactively addressed an internal Corrective and Preventive Action (CAPA) related to six historical Suspected Unexpected Serious Adverse Reaction (SUSAR) submissions for Maralixibat. These events occurred between 2013-2015, prior to Mirum assuming sponsorship, and were identified during the company's internal safety data review in early 2021. The expedited safety reports were subsequently distributed on May 10, 2021, before the inspection's close. Minor protocol deviations, such as a physician not on a delegation log, were noted but addressed at the site level or through internal company procedures. Consequently, no new regulatory actions or specific requirements were imposed by the FDA as a result of this inspection.