Case Study: Italfarmaco achieves FDA approval for Givinostat with Certara’s pharmacometric and regulatory support

Optimizing Dosing and Regulatory Outcomes in Duchenne Muscular Dystrophy (DMD) Drug Development

The pharmaceutical company Italfarmaco developed Givinostat, a treatment for Duchenne muscular dystrophy (DMD), but faced significant challenges due to the rare disease's small patient population. This made trial recruitment, design, and pediatric dosing optimization difficult. Italfarmaco partnered with vendor Certara to implement a model-informed drug development approach, leveraging their pharmacometric services to overcome these hurdles for regulatory submission.

Certara provided essential pharmacometric analysis and regulatory strategy support. This included conducting population PK/PD modeling to refine a weight-based dosing regimen and facilitating discussions with the FDA and EMA. As a result, Certara helped Italfarmaco secure FDA approval for Givinostat, establishing a safe and effective dosing strategy and paving the way for the treatment's expansion to younger patient populations.

Italfarmaco