A population PK model for eculizumab, which had been previously constructed for adult PNH patients, was repurposed to determine dosing for aHUS patients. The mAb’s PK/PD relationship in PNH was also leveraged to determine drug exposure and inform pediatric aHUS dosing.

Blood cells
Drug Development

Two small Phase II studies and a retrospective observational study were conducted, involving just 57 aHUS patients. PK/PD modeling of those data was used to determine the dose- response-effect relationships for two surrogate endpoints. Then, trial simulations based on that modeling were used to determine the best dosing for pediatric and adult patients with aHUS.

Patients with aHUS who were treated with eculizumab had improved platelet counts and other blood parameters and better kidney function with some patients no longer requiring plasmapheresis. The FDA approved eculizumab to treat both adult and pediatric aHUS patients.

FDA Approved

Trial simulations were used to determine the best dosing for pediatric and adult aHUS patients.

Headquartered in the U.S., our client has been a leader in the discovery, development, and commercialization of medicines for rare diseases for nearly 30 years. Its research efforts focus on novel molecules and targets in the complement cascade, and it operates in more than 50 countries.

Our Client


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