Population Pharmacokinetic Modeling to Support Trofinetide Dosing for the Treatment of Rett Syndrome.

ABSTRACT:

Introduction

Oral trofinetide administered using a body weight-banded dosing regimen was approved in the US for the treatment of Rett syndrome (RTT) in patients aged ≥ 2 years. This approval was principally based on efficacy and safety findings of the phase 3 LAVENDER study in girls and women aged 5-20 years with RTT and extended to younger children aged 2-4 years with supporting data from the DAFFODIL study. Weight-banded dosing regimens were selected based on early clinical population pharmacokinetic (popPK) modeling and different scenario simulations. We report the development and application of an updated popPK model to confirm that steady-state trofinetide exposures achieved in individual patients in the LAVENDER study were within target exposure range.

Methods

A previo

SUBMITTER: Darwish M 

PROVIDER: S-EPMC11787235 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature