Alexion's Soliris Gets Orphan Drug Designation For Treating Myasthenia Gravis

By RTT News, 
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(RTTNews.com) - The U.S. Food and Drug Administration or FDA has granted orphan drug designation to Alexion Pharmaceuticals Inc.( ALXN )'s Soliris (eculizumab) for treating patients with Myasthenia Gravis or MG, a rare type of neurologic disorder caused by uncontrolled complement activation.

Soliris or eculizumab is a first-in-class terminal complement inhibitor and is presently approved for the treating patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, rare kind of life-threatening disorders caused by chronic uncontrolled complement activation. However, Soliris is not approved anywhere for treating MG. The company is enrolling patients in a multinational, placebo-controlled registration trial of eculizumab in patients with refractory generalized MG.

Orphan status is specifically granted to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect less than 200 thousand people in the U.S. The designation provides a period of marketing exclusivity for the drug, if regulatory approval is ultimately received for the designated indication.

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