FDA approves first GMG treatment in more than 60 years
Myasthenia gravis is a progressive autoimmune disease that leads to muscle weakness. This can range from eyelid drooping to severe and debilitating symptoms where respiratory and skeletal muscles are affected. As the disease becomes more severe and more muscles become affected, it is referred to as generalized myasthenia gravis (GMG).
Although there are several treatments available for GMG, they do not work for everyone who suffers from the disease, and the recent approval of Soliris in the US is particularly significant for the 10–15% of sufferers who do not respond to available treatments.
These patients are anti-acetylcholine receptor (AchR) antibody-positive, and suffer from serious symptoms that can lead to hospitalisations, and can be life-threatening in the most severe cases.
An effective, long-overdue approach
Soliris is a first-in-class drug that targets and inhibits the complement system, and has been approved specifically for AchR antibody-positive patients. For these patients it is overactivation of the complement system that causes the disease, and plays a particular role in the most severe symptoms. Soliris has been shown to be effective in reducing the symptoms of GMG for these patients, and greatly improves quality of life.
Having seen no new drug approvals in six decades, the GMG market had nothing to offer AchR antibody-positive patients. For sufferers who have been left without a treatment option because of their disease status, this new approval could be a huge turning point.
30 October 2017http://www.pharmaceutical-technology.com/