Alexion Pharmaceuticals reported that the European Commission has designated its drug Soliris (eculizumab) orphan drug status for the treatment of rare disease Myasthenia Gravis (MG).
Soliris is a first-in-class inhibitor of terminal complement, currently indicated for the treatment of two ultra-rare disorders. Both paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) are disorders rooted in chronic and uncontrolled complement activation. The drug received approval for the treatment of these conditions in the U.S., EU, Japan, and other countries.
Now, the EC has conferred orphan status to the drug in MG, a rare and debilitating neurologic disorder. MG is caused by auto-antibodies targeting the nerve muscle junction, resulting in life- long uncontrolled terminal complement activation. The disease is characterized by progressive weakening of various muscle groups in the body, including limbs, ocular, and respiratory muscles.
Dr. Martin Mackay, EVP of Global Head of R&D at Alexion, 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.”
Orphan drug status brings with it a range of incentives for the sponsor company including period of market exclusivity upon approval. Alexion is currently admitting patients with refractory generalized MG for enrollment in a multi-national, placebo controlled registration trial of Soliris.
The company also announced key appointments to its board of directors this month. David Brennan, former CEO and Executive Director of AstraZeneca, Michele Burns, current Center Fellow and Strategic Advisor at Stanford University Center on Longevity, and Christopher J. Coughlin, Senior Advisor, at McKinsey & Co., were named new members of Alexion’s board.