Soliris Drug Granted Orphan Drug Designation By FDA

Alexion Pharmaceuticals announced that the FDA has granted the company’s Myasthenia Gravis (MG) drug Soliris (scientific name eculizumab) orphan drug designation. MG is a rare but serious and debilitating neurologic condition. Patients with MG suffer from uncontrolled complement activation which occurs because of antibodies directed at the neuromuscular junction.

Martin Mackay, PhD, executive vice president, and global head of research and development at Alexion, commented on the MG condition and Alexion’s treatment. “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,” said Mackay. “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 works by inhibiting MG-friendly pathways, and the drug is classified as a terminal complement inhibitor. The drug is currently approved for the treatment of paroxysmal nocturnal hemoglobinuria as well as atypical hemolytic uremic syndrome. Those two conditions are also caused by chronic uncontrolled complement activation. Alexion is currently enrolling patients in a multinational , placebo-controlled trial of eculizumab in patients with refractory generalized MG.

The orphan drug designation will allow Alexion to take advantage of certain benefits, such as the longer period of marketing exclusivity if and when the drug is approved by the FDA and hits the market. Orphan drug designation is usually granted to drugs that treat rare diseases or disorders that affect less than 200,000 patients in the US.