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New Data Showcases Long Term Use of Eculizumab in Myasthenia Gravis

SEPTEMBER 14, 2017
Jim Radke
Data from an open-label extension study involving the use of eculizumab was on full display at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting in Phoenix, Arizona.

The new data shows the drug to be safe and effective to treat patients with myasthenia gravis.

Myasthenia gravis is an autoimmune neuromuscular disease in which antibodies suddenly present and block acetylcholine receptors at the neuromuscular junction. Sibns most commonly include muscle weakness in the eyeballs and eyelids. In more severe forms, symptoms can include weakness of the head, neck, trunk, limb, and respiratory muscles.

The condition is most frequently treated with acetylcholinesterase inhibitors, but in people with refractory myasthenia gravis, other options are often required. One possible treatment option in development is eculizumab, an antibody directed toward the complement system that has already been approved for paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

The open label study (ECU-MG-302) was for patients who completed the 26-week, double-blind, placebo-controlled REGAIN study which tested the use of eculizimab in patients with anti-acetylcholine receptor refractory generalized myasthenia gravis. In the open label extension, patients received eculizumab (maintenance dose: 1200 mg every 2 weeks).

The study found that patients who took eculizimab for 26 weeks in the REGAIN study and then continued to take the drug in the extension study (n=56), had stable scores on their myasthenia gravis activities of daily living (MG-ADL) questionnaire. Further, those switching from placebo to eculizumab (n=60) showed a statistically significant improvement in their scores within 1 week of treatment.

From week 8 through week 52, they maintained a new baseline score indicating sustained improvement in daily living following the introduction of exulizimab.1

Other assessments, including Quantitative MG (QMG), MG Composite (MGC), and MG quality of life (MG-QoL15) also showed patients taking had significant improvement in all scores when switching from placebo to eculizimab while those continuing to take eculizimab maintained their improved scores over 26 and 52 weeks2,3.

References
  1. Howard J, Wang JJ, O’Brien F, Mantegazza R. Efficacy of eculizimab on myasthenia gravis-activities of daily living and its respiratory, bulbar, limb, and ocular domains in patients with AChR+ refractory generalized myasthenia gravis. Presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting; Phoenix, Arizona; September 13-16, 2017. Abstract #210.

  2. Howard J, Wang JJ, O’Brien F, Mantegazza R. Efficacy of eculizumab in maintained beyond 26 weeks in patients with AChR+ refractory generalized myasthenia gravis. Presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting; Phoenix, Arizona; September 13-16, 2017. Abstract #211.
  3. Howard J, Wang JJ, O’Brien F, Mantegazza R. Efficacy of eculizumab sustained over 52 weeks in patients with AChR+ refractory generalized myasthenia gravis: interim results from the open-label extension of REGAIN. Presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting; Phoenix, Arizona; September 13-16, 2017. Late-breaking abstract.


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