Case Study: Ionis Pharmaceuticals accelerates Donidalorsen BLA submission with Certara exposure-response modeling

Supporting Ionis’ Donidalorsen BLA with Biomarker​ HAE Attack Rate Modeling

Ionis Pharmaceuticals faced the challenge of justifying a less frequent dosing regimen for its drug Donidalorsen as part of a Biologics License Application for treating hereditary angioedema. They needed to quantitatively link drug exposure to a biomarker and a clinical efficacy endpoint to meet regulatory expectations and support an accelerated approval pathway. To address this, they engaged Certara's Pharmacometrics services.

Certara developed and validated an exposure-response model using clinical trial data, which established the relationship between the drug, the biomarker prekallikrein, and the rate of HAE attacks. Their analysis and simulations demonstrated that a less frequent dosing schedule could maintain efficacy. This robust modeling strengthened the BLA submission for Ionis, helping them to accelerate their timeline, offer more flexible dosing options for patients, and minimize regulatory delays.

Ionis Pharmaceuticals