This month, Soliris®, a treatment produced by Alexion Pharmaceuticals, Inc. was approved by the European Commission for the treatment of Refractory Generalized Myasthenia Gravis (gMG).
Myasthenia gravis (MG) is a rare, autoimmune, likely antibody-mediated, neuromuscular disease that causes fatigable muscle weakness. Refractory gMG is an ultra rare (about 10-15% of gMG cases), sub-condition of MG. There are multiple treatments for MG that can be used to help manage symptoms, but they can be almost completely ineffective for those patients with refractory gMG.
Refractory gMG not only makes it difficult to do daily activities from showering, to eating, it can also cause extreme health complications like having trouble breathing, leading to its patients ending up in the hospital, or even scarier, the ICU. To learn more about refractory gMG, click here.
Why is this such a critical approval? In the EU, before the approval of Soliris, there we no complement-based therapies for gMG. Soliris® works specifically with patients who have anti-acetylcholine receptor (AChR) antibody-positive refractory gMG. To learn more about how Soliris works for refractory gMG, click here.
In the US, Soliris is already FDA approved for two very rare diseases called paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Approving it for the treatment of refractory gMG could change a lot of US lives. Here’s to hoping the EU’s approval will help move along the approval of Soliris® for refractory gMG in the United States.
