Cerus Corporation (NASDAQ: CERS) announced today that it has submitted an IDE supplement to the U.S. Food and Drug Administration (FDA) for the company’s proposed Phase III trial of the INTERCEPT Blood System for red cells in patients receiving chronic red cell transfusion support for sickle cell disease (SCD) or thalassemia.
“We look forward to working with FDA to finalize the Phase III study protocol to support U.S. licensure of INTERCEPT red cells for support of patients with chronic anemia,” commented Dr. Laurence Corash, Cerus’ chief medical officer. “Chronically transfused patients are at increased risk both from newly emerging pathogens, such as dengue virus, and from known infectious agents, such as Babesia, for which there are no currently licensed tests. Pathogen inactivation of red cells offers a potential new treatment option for this vulnerable patient population, who may face lifetime exposure to blood transfusion.”
The proposed trial design includes approximately 400 patients, who would receive transfusions of INTERCEPT or control red cells for treatment of sickle cell disease or thalassemia. Proposed endpoints of the study are designed to assess both the efficacy and safety of INTERCEPT red cells. FDA has provided guidance that an approximately 60 red cell unit paired in vitro study and an additional Phase I recovery and survival study using the final clinical disposable sets and reagents in healthy subjects will be required prior to initiation of the proposed Phase III trial. These prerequisite studies may need to be finished and the data submitted prior to the FDA’s review of the Phase III protocol supplement filed in the IDE. Previous discussions with the FDA indicated that Cerus’ Phase III protocol could be reviewed under a Special Protocol Assessment (SPA), but upon receipt of the application the FDA amended its position and requested use of the IDE route. The proposed Phase III study size and endpoints in the IDE supplement are identical to those previously planned for SPA review.