The European Commission has granted orphan drug designation to Alexion Pharmaceuticals’ Soliris (eculizumab), a terminal complement inhibitor, to treat patients with myasthenia gravis (MG).
Myasthenia gravis is a neurologic disorder caused by auto-antibodies that recognise a specific target in the nerve-muscle junction, resulting in life-long uncontrolled terminal complement activation that causes tissue damage and interference with signalling between nerve and muscle fibres.
“By specifically inhibiting the terminal complement pathway, we believe that eculizumab has the potential to help patients living with this devastating rare disorder.”
Alexion executive vice president and R&D global head Martin Mackay said: “Patients with MG develop debilitating muscle weakness, impairing their ability to walk, speak clearly, swallow and, in some cases, to breathe normally, which could lead to a life-threatening myasthenic crisis.
“By specifically inhibiting the terminal complement pathway, which is believed to play a pivotal role in the pathophysiology of MG, we believe that eculizumab has the potential to help patients living with this devastating rare disorder.”
Soliris is currently approved in nearly 50 countries for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and in nearly 40 countries for the treatment of atypical hemolytic uremic syndrome (aHUS).
The drug is indicated to inhibit complement-mediated TMA. It is not approved in any country to treat MG.
Alexion is currently enrolling patients in a multinational, placebo-controlled registration trial of eculizumab for people with refractory generalised MG.
The company is evaluating other potential uses for Soliris in addition to treating the severe and ultra-rare disorders PNH and aHUS.