FDA Approves Soliris for Generalized Myasthenia Gravis

The US Food and Drug Administration has approved eculizumab (Soliris) for particular adult patients with generalized myasthenia gravis (gMG).

Soliris, Alexion Pharmaceuticals’ intravenous monoclonal antibody that functions as a terminal complement inhibitor, is used outside of its trade name to treat paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. It has been approved by the FDA for adult gMG patients who are acetylcholine receptor (AchR) antibody-positive.

Patients with the neuromuscular gMG can suffer from difficulties seeing, walking, breathing, talking, and swallowing. Soliris showed efficacious treatment for gMG patients who had failed previous immunosuppressive therapies in the randomized, double-blind, placebo-controlled, multicenter, phase 3 REGAIN study.

These patients, at risk of disease exacerbations and hospitalization-required issues, make up 5 to 10% of the myasthenia gravis patient population. The immune system’s complement system is chronically activated in patients, creating life-threatening complications in gMG patients who are anti-AchR antibody-positive. Soliris combats this mechanism of the disease by inhibiting the terminal complement cascade.

John Orloff, MD, executive vice president and head of Research & Development at Alexion, said the therapy’s approval is a “significant milestone” for both the company and the patient population that continues to suffer from unresolved disease symptoms.

“We are proud that we could apply our deep understanding of complement biology to develop Soliris for the treatment of patients with this debilitating neuromuscular disorder,” Orloff said.

Nancy Law, chief executive officer of the Myasthenia Gravis Foundation of America (MGFA), echoed that it is a “landmark day” for a community that has not seen a gMG therapy approved in more than 60 years.

“It is particularly significant that this approval of Soliris will provide a new option for those with gMG and especially for those who do not respond adequately to or cannot tolerate standard treatment options,” Law said.

Soliris was previously approved for the patient population in the European Union, and has received Orphan Drug Designation for the treatment of patients with myasthenia gravis in the US and Europe.