The drug was approved in the EU and US. The dosage of 0.3 mg/kg was approved and listed on the label.
An investigational drug was in Phase 3 to treat a rare genetic disease disorder. The number of subjects participating in the Phase 3 study was limited. Phase 3 study demonstrated clinically significant efficacy and favorable safety. The client is preparing for an NDA. Although 0.3 mg/kg was used for all patients, it was unknown whether this dose could be justified in the NDA submission.
XP Pharma proposed an analysis plan. We divided patients into four quartiles based on pharmacokinetic (PK) exposures. The efficacy endpoint was analyzed for each quartile. We found that efficacy was comparable across the 4 PK-exposure quartiles, indicating that the PK exposures at 0.3 mg/kg reached the plateau of the dose-response curve. Likewise, the frequency of adverse events (AEs) did not increase with increasing PK exposure to the drug, indicating that the incidence of these AEs was independent of the PK concentrations of the three analytes. The analysis results were included in the CTD 2.7.2 summary of Clinical Pharmacology for NDA and other CTD modules.
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