The vaccine is expected to enter phase II human trials in hematopoietic cell transplant (HCT) donor-recipients in the second half of 2005.
The Orphan Drug Act is intended to encourage development of products for rare diseases affecting fewer than 200,000 people in the US. For products designated as orphan drugs, FDA will not approve other applications to market the same product for the same indication for a period of seven years after product approval.
In addition to potential market protection, orphan-drug designation provides certain tax credits, opportunities for regulatory agency assistance on review of clinical protocols, and waiver of the Prescription Drug User Fee Act (PDUFA) filing fee.
Cytomegalovirus (CMV) infection is predicted to affect up to 60% of the estimated 29,000 patients undergoing HCT or solid organ transplant (SOT) procedures in the US annually, causing transplant rejection, serious illness and even death if untreated. Expensive antiviral drug therapy is currently used to control the disease, but does not eliminate the infection.