Gamida Cell is developing StemEx in a joint venture with Teva Pharmaceutical Industries. The design of the study was submitted under a special protocol assessment.
“The design and planned analysis of your study sufficiently address the study’s objectives,” the FDA stated.
A phase I/II study of StemEx showed safety and trends of efficacy. StemEx was granted an FDA orphan drug designation in March 2005.
“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,” said Gamida Cell CEO.
Stem cell transplantation is a life saving procedure for patients with high-risk hematological malignancies. Currently, many patients lack a suitable marrow donor. StemEx may provide an alternative source of stem cells for these patients by enabling the use of cord blood for transplantation.