Northfield Laboratories Inc has reached agreement with the U.S. Food and Drug Administration (FDA) on Special Protocol Assessment (SPA) for its pivotal Phase III prehospital trial with PolyHeme, the company's oxygen-carrying blood substitute.

"This is an exceptional achievement for Northfield. The SPA confirms that the data from our landmark trial will form the primary basis for an efficacy claim in a Biologics License Application for PolyHeme," said Steven A. Gould, Chairman and Chief Executive Officer. "SPA is not a routine event and this is not a routine trial. SPA is particularly useful for studies which assess novel, potentially life-saving therapies, especially when no approved drug is available to address a critical, unmet clinical need. Since agreement on SPA has only occurred in the Center for Biologics Evaluation and Research (CBER) on a limited number of occasions, this is a true milestone."

SPA represents acknowledgement and confirmation of a mutual agreement between a clinical trial sponsor and FDA that successful completion of the proposed trial will form the primary basis for an efficacy claim as part of product approval. SPA documents agreement on the design, primary efficacy endpoints, study conduct, data analysis, and the kind of labeling statements that could be made if the data are supportive and the product is approved. The agreement becomes part of the administrative record and may only be changed by mutual agreement of the parties, or if FDA identifies a substantial scientific issue relevant to safety or efficacy after the trial has begun.

"During the past year, we have been pursuing a regulatory strategy designed to reduce uncertainty and improve predictability by taking advantage of FDA regulations specifically intended to expedite the approval of novel therapies such as PolyHeme. SPA is the cornerstone of that strategy, and as we move towards licensure, this agreement provides the regulatory clarity that will facilitate our efforts to seek fast track designation for PolyHeme as a product intended to treat life-threatening blood loss when blood is unavailable," added Dr. Gould.

The PolyHeme trial is designed to assess the safety and efficacy of PolyHeme in improving survival when used to treat severely injured and bleeding trauma patients beginning at the scene of injury and continuing during ambulance transport to the hospital. Because the patients eligible for the study are unlikely to be able to provide consent due to the extent and nature of their injuries, the trial will be conducted under federal regulations that allow clinical research in emergency settings using an exception from the requirement for informed consent.

Northfield anticipates that approximately 20 Level I trauma centers throughout the United States will eventually participate in the PolyHeme trial, which has an expected enrollment of 720 patients. The process of public disclosure and community consultation required under the regulations is underway at a number of potential trial sites across the country. Patient enrollment will begin as soon as local IRBs complete their review process.