Rare Disease Report

FDA Accepts Alexion's sBLA for Generalized Myasthenia Gravis

MARCH 08, 2017
Andrew Black
Some good news from Alexion for the Myasthenia Gravis (MG) community as the FDA has accepted their supplemental Biologics License Application (sBLA) to extend the indication for Soliris for the treatment of refractory generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody-positive.
Alexion filed the sBLA following data from their Phase 3 study which showed the drug was somewhat effective, but overall failed to meet its primary endpoint.

About the trial

The study was a randomized, double-blind, placebo-controlled, multicenter trial evaluating the safety and efficacy of Soliris (eculizumab) in patients with refractory generalized myasthenia gravis (n=125).
Patients were randomized to receive eculizumab or placebo for 26 weeks. Patients initially received 900 mg of eculizumab weekly for 4 weeks followed by 1200 mg of eculizumab one week later, and then 1200 mg of eculizumab every two weeks.
In the study, the primary efficacy endpoint of change from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score, a patient-reported assessment, at week 26, did not reach statistical significance (P = .0698) as measured by a worst-rank analysis.
Last summer, data from the study was presented at the International Congress on Neuromuscular Diseases (ICNMD) in Toronto, Canada and numerous other outcome measures indicated that the drug was effective. These included changes in Myasthenia Gravis Composite score at week 26, change in the 15-item Myasthenia Gravis Quality of Life.
The FDA must have taken this into consideration during their review process. 

About Soliris 

Soliris (eculizumab) is a complement inhibitor currently approved to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and patients with atypical hemolytic uremic syndrome (aHUS).

About Myasthenia Gravis

In myasthenia gravis, antibodies block, alter, or destroy the acetylcholine receptors at the neuromuscular junction. The condition is usually treated with acetylcholinesterase inhibitors that can increase the concentration of acetylcholine at the neuromuscular junction

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