A prospective observational post authorization safety sub-registry study to characterize the long-term safety profile of eliglustat in adult patients with Gaucher disease - Sanofi

About this study

Eliglustat is a medication used to treat Gaucher disease, a rare genetic condition where fatty substances build up in organs, like the liver, spleen, and bones. This study collected long-term safety information about patients who started taking the medicine after it was approved for use. 

Data accessed by Sanofi included population level information, diagnosis details, and records of adverse events. The safety monitoring that took place in the study is required by health authorities to ensure the medication remains safe for patients over time. Sanofi carried out the study at up to 20 centres across Europe and the United States, including the UK.

The primary goal of the study was to assess the long-term safety of eliglustat in real-world patients, by evaluating how common adverse events were. Additionally, it seeks to describe the real-world use of the drug and how well it benefits the Gaucher disease patient population.