Pharmaceutical trial for eculizumad (Soliris) announced for patients with mysathenis gravis and neuromyelitis

Cheshire Connecticut - April 24 2014 -  TAlexion Pharmaceuticals (Nasdaq:ALXN) today announced the initiation of a single, multinational, placebo-controlled trial to evaluate the safety and efficacy of eculizumab (Soliris®) in patients with relapsing neuromyelitis optica (NMO), a life-threatening, ultra-rare neurologic disorder. Alexion also initiated a single, multinational, placebo-controlled trial in patients with refractory generalized myasthenia gravis (MG), another rare and debilitating neurologic disorder.


“We look forward to enrolling patients in these placebo-controlled studies to confirm the clinical benefits of eculizumab in the treatment of NMO and MG, which would be an important development for these underserved patient populations.”

Both NMO and MG are disorders caused by uncontrolled complement activation. In patients with NMO, chronic, uncontrolled complement activation results in severe damage to the central nervous system (CNS), predominantly impacting the optic nerve and spinal cord. This devastating disease is characterized by severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. Currently, there are no approved treatments for NMO. In patients with MG, uncontrolled complement activation due to antibodies directed at the neuromuscular junction can ultimately lead to profound and debilitating weakness of various muscle groups throughout the body. Patients with generalized MG develop significant muscle weakness, impairing their ability to walk, speak clearly, swallow and, in some cases, to breathe normally.

“Since complement activation plays a pivotal role in the pathophysiology of both NMO and MG, the mechanism of action of eculizumab, as a terminal complement inhibitor, suggests it may have the potential to help patients living with these rare and devastating disorders,” said Martin Mackay, Ph.D., executive vice president and global head of R&D at Alexion. “We look forward to enrolling patients in these placebo-controlled studies to confirm the clinical benefits of eculizumab in the treatment of NMO and MG, which would be an important development for these underserved patient populations.”

Soliris is currently approved in the United States, European Union, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is not approved in any country for the treatment of NMO or MG.

Continue >>> About The Studies