Background & Problem
The Sponsor’s drug had been approved in Europe for decades, but had not been developed in the US. The Sponsor wanted to bridge the studies that were conducted in Europe with a few additional studies conducted in the US to gain approval of the drug by the FDA. Since it had been quite a while since it was approved in Europe, pharmacokinetic (PK) data were lacking.
The Sponsor enlisted our help to design a strategy that would bridge between the current study and the previous clinical experience and to help guide clinical trial design in upcoming studies.
Nuventra’s scientists developed a population PK model based on data from a current pediatric study and created a exposure/dose/response model to simulate which doses and which dose regimens would likely prove efficacious and remain safe in upcoming studies.
Simulations indicated that a certain dose range in patients between 2 and 5 years old and doses up to a certain amount in non-ambulatory patients would remain safe.