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

Patients

December 2024

Summary

An investigation into the long-term safety of the drug eliglustat in adults with Gaucher disease.

This study, run by Sanofi, sought to gather data on patients being treated for Gaucher disease, to monitor the ongoing effectiveness of their licensed medicine. 

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 through the International Collaborative Gaucher Group (ICGG) Gaucher registry 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, 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. 

Data sources used:

• Longitudinal secondary care data: information from patients who received care in specialist clinics over an extended period of time

• International Collaborative Gaucher Group (ICGG) Gaucher registry: information on Gaucher disease patients collected across different countries.

Benefits of the study

By accessing data from patients receiving treatment for Gaucher disease across multiple countries through the ICGG Gaucher registry, this Sanofi study was able to confirm the long-term safety and effectiveness of the treatment. 

Pharmaceutical companies are required to monitor the safety of their treatments, once approved for use, to ensure long-term benefits and identify potential risks to the patient population. By allowing access to the required data, companies can ensure local health systems, regulators and the general public have evidence of the benefits of treatment they prescribe.  

Further information

Read the clinical study

Glossary

Observational post-authorization safety sub-registry study: research conducted after a medicine is approved. It collects long-term safety data from patients using the medicine in normal healthcare settings, without changing their treatment.

Safety profile: summary of all the known side effects and risks associated with a medicine.

Real-world patients: patient data collected in a typical healthcare environment, such as a hospital or GP surgery, rather than in a clinical trial.

Last modified: 15 February 2025

Last reviewed: 15 February 2025