EU Regulators Give Alexion’s Soliris Orphan Drug Status

European regulators have granted Alexion Pharmaceuticals’ blood disease therapy Soliris orphan drug designation for the treatment of myasthenia gravis, putting the company on its way toward a third indication for the product.

Soliris (eculizumab) already is approved in nearly 50 countries for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, two extremely rare and life-threatening blood disorders. Alexion has yet to receive approval anywhere for myasthenia gravis, a rare neurological disorder that can lead to debilitating weakness of various muscle groups throughout the body.

The company is now conducting a Phase III trial for the indication at 21 sites in the U.S., Canada and Japan. Should the drug win EU approval for myasthenia gravis, it would receive an extended period of marketing exclusivity based on its orphan drug designation, Alexion said.

Other treatments for myasthenia gravis, such as cholinesterase inhibitors and immunosuppressant medications, may help some patients, but not all respond and some respond only within four to eight months, according to the European Federation of Pharmaceutical Industries and Associations (EFPIA).

The European Commission grants orphan drug status to therapies for conditions affecting no more than five in every 10,000 people in the EU. EFPIA estimates that roughly 15 out of every 100,000 people in the EU live with myasthenia gravis, representing approximately 72,000 cases. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.