Friday, June 20, 2014

Clinical Trial Of Soliris Starting For Myasthenia Gravis Patients

The FDA (Food and Drug Administration) has allowed orphan drug status for Alexion Pharmaceuticals' Soliris (eculizumab) for treating patients with Myasthenia Gravis (MG). Myasthenia Gravis a rare, debilitating neuromuscular disorder brought on by uncontrolled complement activation. In patients with MG, such 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.

Alexion announced it is currently enrolling patients
in a multinational, placebo-controlled registration trial of Soliris in patients with refractory (uncontrolled) generalized myasthenia gravis. More information on this trial is available at and listed under the identifier NCT01997229.

Soliris is a complement inhibitor and was first approved in 2007 to treat patients with paroxysmal nocturnal hemoglobinuria (PNH). The drug was also approved in 2011 to treat patients with atypical hemolytic uremic syndrome (aHUS).

Soliris was granted orphan drug designation in January 2014 to prevent delayed graft function in renal transplant patients. DGF is an early and serious complication of organ transplantation that is characterized by the failure of a transplanted organ to function normally immediately following transplantation.

The FDA, through its Office of Orphan Products Development), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

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