Gamida Cell's StemEx® Registration Study Receives Special Protocol Assessment from the FDA
Gamida Cell Ltd. has announced that it has reached an agreement under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA) on the design of the global, pivotal, historical controlled registration study of StemEx® for the treatment of hematological malignancies.
Gamida Cell is developing StemEx® in a Joint Venture with Teva Pharmaceutical Industries Ltd.
In response to the SPA application, the FDA stated in an official reply letter that "...the design and planned analysis of your study sufficiently address the study’s objectives and that this study is adequately designed to provide the necessary data that, depending upon outcome, could support a license application submission."
"We have worked hard, together with the FDA, to sculpt the design and analysis of the study," said Gamida Cell CEO Dr. Yael Margolin.
"Conducting a pivotal study under an SPA is an important achievement. Among other advantages, it provides an agreed upon roadmap for the management and analysis of the study's data, and thus reduces some of the risks involved in seeing a product to market."
"Assuming that all of the study endpoints are met, we are confident that, together with Teva, we will be able to bring StemEx® to the market in 2009 as planned."