Alexion’s rare disease therapy Soliris can now be used in the US to treat generalised myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AchR) antibody-positive, offering this group the first new treatment option in more than 60 years.

Myasthenia gravis is a debilitating, chronic and progressive autoimmune neuromuscular disease. It typically begins with weakness in the muscles that control the movements of the eyes and eyelids, and often progresses to the more severe and generalised form - gMG- with weakness of the head, neck, trunk, limb and respiratory muscles.

In patients with anti-AchR antibody-positive MG, the body’s own immune system turns on itself to produce antibodies against AchR, a receptor located on muscle cells at the neuromuscular junction and used by nerve cells to communicate with the muscles these nerves control.

Patients with anti-AchR antibody-positive gMG who continue to suffer from severe disease symptoms and complications despite current therapies represent around 5-10 percent of all patients with MG and urgently need new treatment options.

Soliris is a first-in-class complement inhibitor that works by inhibiting the terminal part of the complement cascade, thus targeting a critical underlying cause of the disease.

In clinical trials, the therapy induced treatment benefits for patients who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms, which can include difficulties seeing, walking, talking, swallowing and breathing.

“It is exciting that patients who have not responded adequately to existing therapies will now have a new treatment option that was shown in clinical studies to improve patients’ symptoms, their ability to carry out activities of daily living and their quality of life,” said professor James Howard, Department of Neurology at the University of North Carolina, Chapel Hill, and lead investigator in the clinical development of this new indication.

Soliris is already approved in the European Union for refractory gMG in adults who are anti-AchR antibody-positive, as well as for patients with the ultra-rare disorders paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).