Modeling and simulation has played an important role in supporting regulatory approvals of caplacizumab for treatment of acquired thrombotic thrombocytopenic purpura (aTTP). For example, in 2018 the EMA agreed that clinical studies of caplacizumab in the pediatric population are not feasible. Alternatively, a modeling and simulation approach was applied to identify an appropriate dosing strategy in children. As a result, in Europe, a pediatric label extension was approved in 2020.
The modeling and simulation study at the basis of this label extension is described in this recent publication.