Efgartigimod is a novel investigational medicine for the treatment of chronic inflammatory demyelinating polyneuropathy(CIDP). It is a humanized monoclonal antibody that binds to the neonatal Fc receptor(FcRn) and inhibits its function. FcRn is a protein that is expressed on the surface of many cells, including immune cells. It is responsible for the recycling of antibodies and other proteins, which helps to maintain their levels in the body. By inhibiting FcRn, efgartigimod reduces the levels of antibodies in the body, including those that are involved in the pathogenesis of CIDP.
Open-Label Extension Study
An open-label extension study is a type of clinical trial in which patients who have completed a previous clinical trial of an investigational medicine are given the opportunity to continue taking the medicine for a longer period of time. This type of study is used to collect additional safety and efficacy data on the medicine.
The open-label extension study of efgartigimod for CIDP included 67 patients who had completed the previous phase 2 clinical trial of the medicine. The patients were treated with efgartigimod for up to 2 years.
Results
The results of the open-label extension study showed that the benefits of efgartigimod were sustained in patients with CIDP for up to 2 years. The primary endpoint of the study was the proportion of patients who achieved a meaningful improvement in their symptoms, as measured by the Inflammatory Neuropathy Cause and Treatment(INCAT) score. The INCAT score is a validated measure of disease severity in CIDP.
At the end of the study, 82% of patients had achieved a meaningful improvement in their INCAT score. This was similar to the proportion of patients who had achieved a meaningful improvement in the previous phase 2 clinical trial.
The safety and tolerability of efgartigimod were also assessed in the open-label extension study. The medicine was generally well-tolerated, with the most common side effects being infusion-related reactions, such as chills, fever, and nausea.
Conclusion
The results of the open-label extension study provide further evidence of the long-term benefits of efgartigimod in patients with CIDP. The medicine was well-tolerated and was associated with a sustained improvement in symptoms over a period of up to 2 years.
Further Research
Additional research is needed to confirm the long-term benefits of efgartigimod in patients with CIDP. This includes studies to evaluate the medicine’s efficacy and safety in larger populations of patients and over longer periods of time.
Kind regards
F. Hillsom
