Newly reported data from a clinical trial led by a UNC School of Medicine researcher show that eculizumab may be helpful in treating treating refractory generalized myasthenia gravis (gMG), an extremely rare neuromuscular disorder characterized by weakness and fatigue of skeletal muscles. It has no FDA-approved treatment.
James F. Howard Jr., MD, Distinguished Professor of Neuromuscular Disease, professor of neurology, medicine and allied health, and chief of the Neuromuscular Disorders Section in the UNC School of Medicine, presented the results July 7 at the 14th International Congress on Neuromuscular Diseases (ICNMD) in Toronto.
“Although the REGAIN study narrowly missed its primary endpoint, the additional data presented today suggest a magnitude of effect of eculizumab in refractory MG patients across four separate scales of disease severity that is unprecedented in my clinical investigation experience.
"These findings are particularly meaningful given the urgent need for a first-ever therapy with the potential to have a transformative impact on patients with refractory MG, who continue to face disabling limitations in their daily lives," said Howard, who was the study's principal investigator.
Eculizumab is manufactured by Alexion Pharmaceuticals, Inc. and sold under the name Soliris®. To read Alexion's press release about the study, click here.