Dorota Gliwicz- Miedzinska – FDA EIR, Jun 18, 2021

An FDA pre-approval inspection was conducted at the clinical investigator site of Dr. Dorata Gliwicz-Miedzinzka, MD, in Warszawa, Poland, from June 14-18, 2021. This was a data-validation foreign inspection, conducted under CPGM 7348.811 for Clinical Investigators, focusing on a Mirum Pharmaceuticals clinical study (Protocol LUM0001-304) supporting NDA214662/IND 119917, regulated under §505 of the Federal Food, Drug, and Cosmetic Act. The inspection aimed to verify data from a long-term, open-label study involving patients with Alagille Syndrome.

The review covered informed consent, medical histories, lab results, adverse event reporting, and investigational product control for two enrolled subjects. No Form FDA 483, Inspectional Observations, was issued, indicating no significant violations. Minor observations included occasional gaps in eDiary data reporting, one isolated transcription error in lab results for a single subject, and issues with investigational product shipments lacking temperature monitoring devices. The sponsor provided conversion factors for some lab values and shipping container validations to address concerns. All protocol deviations were accurately reported to the sponsor.

During the close-out meeting, the investigator discussed the minor deviations, particularly emphasizing the need for documenting discussions with the study sponsor regarding investigational product shipments. No formal required actions were mandated by the FDA at the conclusion of the inspection.