DOR BIOPHARMA SUBMITS EUROPEAN APPLICATION FOR GVHD DRUG

DOR BioPharma announced that it has submitted a marketing authorization application to the European Medicines Evaluation Agency for orBec (oral beclomethasone dipropionate) for the treatment of gastrointestinal graft-versus-host disease (GI GVHD), the most common life-threatening complication of allogeneic hematopoietic stem cell transplantation in cancer patients. The application will be reviewed under the centralized licensing procedure, which, if approval is granted, provides a marketing license valid in all 25 member states of the EU.

OrBec is a two-tablet system containing the highly potent, topically active corticosteroid beclomethasone dipropionate, and is designed to specifically target and treat upper and lower GI GVHD with reduced systemic immunosuppressive side effects. Systemic immunosuppressive agents such as prednisone, which are the current standard treatments for GI GVHD, are associated with high mortality rates due to infection and debility. Further, these drugs have not been approved for treating GI GVHD in the EU or in the U.S., but are rather used off-label as investigational therapies for this indication.

The data provided in the submission demonstrate that orBec safely provides a higher rate of survival when compared with the current standard of care, and a lowered exposure to systemic corticosteroids following allogeneic transplantation. The filing is supported by data from two randomized, double-blinded, placebo-controlled clinical trials. The first trial was a 129-patient pivotal Phase III multicenter trial conducted at 16 leading bone marrow/stem cell transplant centers in the U.S. and France. The second trial was a 60-patient Phase II single-center clinical trial conducted at the Fred Hutchinson Cancer Institute. Although orBec did not achieve statistical significance in the primary endpoint of its pivotal trial, namely time to treatment failure through day 50, the treatment did achieve statistical significance in other key outcomes such as median time to treatment failure through day 80, and it demonstrated a statistically significant long-term survival advantage compared with placebo.