Regulatory agency agreed with the results of the integrated assessments conducted by XP Pharma. Client obtained the waiver for conducting a standalone TQT study.
XP Pharma was asked to evaluate the risk of QT prolongation for a small molecule. The small molecule drug was being developed for treatment of multiple different types of seizures at phase 3 stage. Usually, for a small molecule at phase 3 stage, a thorough QT/QTc study should be conducted to support NDA. Because a thorough QT/QTc study is expensive and time-consuming, the question was whether the study could be waived based on the available preclinical and clinical data.
To answer this question, XP Pharma proposed a strategy to address this question. Our overall assessment of the risk for QT prolongation included nonclinical data, the time course of QT prolongation, the magnitude of QT prolongation, categorical analyses of outliers, and certain adverse events (AEs) in patients that can signal potential proarrhythmic effects. A total of eight nonclinical studies, ten phase 1 and 2 clinical studies, and three individual and pooled concentration-QTc modeling analyses from multiple single ascending dose and multiple ascending dose studies were included. We authored a 140- page report for the results of the integrated analysis to support client’s application to waive a TQT study.
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