FDA EIR - Sarepta Therapeutics, Inc. - June 23, 2016

Sarepta Therapeutics, Inc. underwent a focused FDA bioanalytical inspection at its Corvallis, Oregon, facility from June 20 to June 23, 2016. This high-priority inspection, requested by the Division of Neurology Products, aimed to evaluate the integrity of western blotting analytical procedures and associated data for Study SR-CR-16-003, an interim pharmacodynamics analysis of the PROMOVI 4658-301 clinical trial for Duchenne Muscular Dystrophy (IND # 077429, NDA # 206488).

The inspection assessed the firm's adherence to blinding procedures, tissue specimen handling, and the conduct of western blot analyses. It covered sample receipt, storage, and processing, along with the development of controls and randomization schemes. Notably, this was not a Good Laboratory Practices or Good Manufacturing Practices inspection.

While no Form FDA 483 (Inspectional Observations) was issued, the inspection concluded with a scientific discussion identifying several areas for improvement. These included the need for a more robust western blotting detection and quantitation method for low dystrophin levels, the implementation of more precise assays like quantitative mass spectrometry, and the development of a more reliable reference standard with less variability for accurate dystrophin quantitation. Sarepta Therapeutics acknowledged this feedback, indicating ongoing efforts to enhance their protein analysis methods and a future proposal for a new cell line-based reference standard.