Orphan status in Europe for Alexion's Soliris in myasthenia gravis

The European Commission has granted orphan drug designation (ODD) to US drugmaker Alexion Pharmaceuticals’ (Nasdaq: ALXN) Soliris (eculizumab) for the treatment of patients with myasthenia gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation.

In patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can ultimately lead to profound and debilitating weakness of various muscle groups throughout the body.

Solaris, which is currently approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation, recently  gained orphan status from the US Food and Drug Administration for the treatment of MG.