Alexion's Soliris Granted Orphan Status By The FDA

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Alexion Pharmaceuticals’ Soliris (eculizumab) for the treatment of rare disease Myasthenia Gravis (MG).

Soliris is a first-in-class terminal complement inhibitor indicated to reduce hemolysis. The drug is currently approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two other ultra-rare diseases caused by chronic uncontrolled complement activation. Soliris has received regulatory approval in the U.S., EU, Japan, and other countries around the world.

MG is a rare neurologic disorder in which auto-antibodies target the nerve-muscle junction, resulting in lifelong uncontrolled terminal complement activation causing damage and signal interference between nerve and muscle fibers. Patients affected with MG suffer weakness in the eye muscles, progressing to weakness in head, limb, trunk, and respiratory muscles.

“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,” said Martin Mackay, Executive Vice President, Global Head of R&D at Alexion.

The company is accepting enrollment in a placebo-controlled, multinational registration study involving patients with refractory generalized MG.

The FDA Office of Orphan Products Development (OOPD) grants orphan status to investigational drugs and biologic products that are being developed for treatment, diagnosis, or prevention of rare diseases or disorders. The drugs must target a disease affecting less than 200,000 people in the U.S. to be granted orphan status. The designation bestows sponsor companies with a range of benefits including exemption from certain FDA fees and a period of marketing exclusivity upon approval of the drug.