FDA OKs Soliris for Generalized Myasthenia Gravis

Officials with Alexion Pharmaceuticals, Inc. have announced that the FDA has approved eculizumab (Soliris) as a treatment for adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.

The FDA based its approval of the extended indication for Soliris on clinical data from the phase 3, randomized, double-blind, placebo-controlled, multicenter REGAIN study (ECU-MG-301). During REGAIN and its ongoing open-label extension study, eculizumab demonstrated treatment benefits for patients with anti-AchR antibody-positive gMG 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. These patients are at an increased risk of disease exacerbations and crises that may require hospitalization and intensive care and may be life-threatening. These patients represent approximately 5-10% of all patients with MG.^1-6

Chronic uncontrolled activation of the complement system, a part of the immune system, plays a major role in the debilitating symptoms and potentially life-threatening complications for patients with gMG who are anti-AchR antibody-positive.^1-4 By selectively and effectively inhibiting the terminal complement cascade, Soliris targets an underlying cause of the disease.

References

1 Silvestri N, Wolfe G. Treatment-refractory myasthenia gravis. J Clin Neuromuscul Dis. 2014;15(4):167-178.

2 Suh J., Goldstein JM, Nowak RJ. Clinical Characteristics of Refractory Myasthenia Gravis Patients. Yale J Biol Med. 2013;86(2):255-260.

3 Howard J. Myasthenia Gravis — A summary. Myasthenia Gravis Foundation of America. http://www.myasthenia.org/HealthProfessionals/ClinicalOverviewofMG.aspx. Accessed October 12, 2017.

4 Grob D, Brunner N, Namba T, Pagala M. Lifetime course of myasthenia gravis. Muscle Nerve. 2008 Feb;37(2):141-9.

5 Souayah N, Nasar A, Suri MF, et al. Int J Biomed Sci. 2009;5(3):209-214.

6 Engel-Nitz N, et al. Poster 146; ICNMD 2016.