this press release says it all
this is why the treatment failure rate at 50 days met statistical significance. The first 10 days of treatment failure before, orbec had a chance to work overweighed the median to the beginning of the trial. by the time you stretch it out to 80 days it shows significance again. The ODAC panel was not made aware of this distinction
Treatment for Intestinal Graft-versus-Host Disease (iGVHD)
DOR BioPharma to File NDA for orBec
MIAMI, April 20, 2005 - DOR BioPharma, Inc. (AMEX: DOR) ("DOR" or the "Company") announced its intention to file a new drug application (NDA) with the FDA for orBec (oral beclomethasone dipropionate) for the treatment of intestinal Graft-versus-Host Disease (iGVHD), a common serious complication of bone marrow transplantation for cancer. The decision follows recent correspondence and a pre-NDA meeting with the FDA.
I think after reviewing the data they can see that at 50 days it worked. It only failed through 50 days because the first 10 days screwed up the median time to treatment failure.
"We are very encouraged by our meeting with the FDA," stated Michael Sember, President and Chief Executive Officer of DOR. "We are excited with the results of our pivotal Phase III clinical trial. In addition to the strong positive trends on our primary endpoint of median time to treatment failure at study Day 50 in our pivotal trial, we saw a statistically significant result in median time to treatment failure at study Day 80 (p-value 0.0226). This result, combined with a highly statistically significant outcome and approximately 70% reduction in the prospectively defined secondary endpoint of mortality at 200 days post transplant (p-value 0.006), has given us confidence in the consistency and clinical durability of activity of orBec. We believe that our pivotal Phase III clinical trial of orBec and collectively all the clinical data before it, clearly demonstrate a clinical benefit to patients with no other alternative except high dose systemic corticosteroids. Our immediate priorities are to assemble a high quality data package to support the filing of an NDA as soon as possible but by the fourth quarter of 2005, and to submit the results of the pivotal trial for publication in a prominent peer-reviewed publication. We will also take steps to prepare for submission of a Marketing Authorization Application (MAA) with the European Medicines Evaluation Agency (EMEA)."
orBec has previously been granted Orphan Drug Designation and has received "Fast Track" designation from the FDA.
George B. McDonald, MD, Head of the Gastroenterology/Hepatology Section at the Fred Hutchinson Cancer Research Center in Seattle, inventor of orBec, and a consultant to DOR stated, "Those working in the field of hematopoietic cell transplantation have long recognized that prolonged exposure to prednisone in patients with iGVHD led not only to weakness, debility, and changes in body habitus, but also to viral, bacterial, and fungal infections. The pivotal Phase III study provides a clear rationale for the use of orBec in the treatment of iGVHD; control of iGVHD with an oral topical corticosteroid leads to less prednisone exposure, less systemic immunosuppression, fewer fatal infections, and possibly an enhanced graft-versus-leukemia (GVL) effect. The frequency of leukemic relapse after allogeneic hematopoietic cell transplant is lower in patients who experience GVHD after transplant, a phenomenon called the GVL effect. We hypothesize that avoidance of prolonged prednisone exposure preserves the GVL effect, resulting in fewer relapses of leukemia. orBec appears to be safe. Thus, the benefit-to-risk ratio for the use of orBec in the treatment of iGVHD seems strongly in favor of benefit. orBec addresses the unmet medical need for an effective adjunctive therapy for iGVHD with minimal side effects."
