FDA GRANTS PRIORITY REVIEW TO ALEXION'S SOLIRIS BLA

Alexion Pharmaceuticals announced that the FDA has accepted the company's biologics license application (BLA) for Soliris (eculizumab) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). In addition, the FDA has informed Alexion that it has granted the application priority review, which targets an FDA action within six months of the submission date. Alexion submitted the BLA in September. Priority review status is granted by the FDA to products that, if approved, would be a significant improvement over existing therapies, according to the company.

The European Medicines Agency has also accepted the Soliris application for review under the agency's accelerated assessment procedure.

The BLA submission is based on data from the pivotal Phase III TRIUMPH trial, which met all prespecified primary and secondary endpoints with statistical significance. Details regarding the TRIUMPH study results were published in the Sept. 21 issue of the New England Journal of Medicine. The BLA also includes interim data of at least six months from the open-label Phase III SHEPHERD safety trial. The last patient completed the last visit in the SHEPHERD trial in October and final 12-month data from the trial will be submitted to the regulatory agencies. The TRIUMPH trial involved 87 PNH patients, and the SHEPHERD trial has enrolled 97 PNH patients.

PNH is an acquired genetic blood disorder characterized by destruction of red blood cells by the body's complement system. Soliris, a long-acting C5 terminal complement inhibitor, is an investigational humanized monoclonal antibody drug designed to selectively block terminal complement activation and thereby restore complement inhibition in the blood of patients with PNH. It has been granted orphan drug status in the by both the FDA and European regulators.