DOR BioPharma Inc. announced that it has received a not approvable letter from the US Food and Drug Administration (FDA) for its lead product orBec (oral beclomethasone dipropionate, or oral BDP) for the treatment of acute gastrointestinal Graft-versus-Host disease (GI GVHD), a common and potentially life-threatening complication of haematopoietic cell transplantation. The FDA has requested data from additional clinical trials to demonstrate the safety and efficacy of orBec. Additionally, the FDA has requested information with respect to other sections of the NDA.
"We are very disappointed that the FDA has deemed orBec not approvable and that additional clinical trials will be required," said Christopher J. Schaber, PhD, president and chief executive officer, DOR. "While we were hoping for a nearer term approval for orBec given the clinical effect we believe we demonstrated in our two randomized, double-blind, placebo-controlled trials, we firmly believe in the pharmacology of orBec and its potential to help address the overwhelming need for a safe and effective treatment for patients suffering from GI GVHD. We will be requesting a meeting with the FDA to discuss this letter and the potential next steps that need to be taken. We will also be reviewing the various business development opportunities we potentially have before us. Once we gain further clarity, we will be able to provide accurate guidance moving forward. We remain focused on our mission to bring this potentially life-saving therapy to the patients who need it as rapidly as possible."
George B. McDonald, MD, head, gastroenterology/hepatology section at the Fred Hutchinson Cancer Research Centre said, "It is disappointing that the FDA has required additional data concerning orBec in view of the clinically compelling data that it has shown in phase II and III clinical trials, both of which demonstrated clear evidence of clinical benefit. These data have convinced many in the transplant oncology community that use of oral BDP should be part of our standard practice in treating gastrointestinal GVHD. Those of us who treat GVHD know what a devastating complication of haematopoietic cell transplantation it really is, even in its less severe forms. Not only are the gastrointestinal symptoms of GVHD unpleasant and persistent, but also the standard treatment using prolonged courses of prednisone leads to debility, serious infections and sometimes death not from GVHD per se, but from complications related to prednisone. Physicians treating GI GVHD need alternative options such as orBec, which has been shown to reduce GVHD symptoms while allowing for reduced use of prednisone."
orBec represents a first-of-its-kind oral, locally acting therapy tailored to treat the gastrointestinal manifestation of GVHD, the organ system where GVHD is most frequently encountered and highly problematic. orBec is a two-tablet system containing the highly-potent, topically-active corticosteroid beclomethasone dipropionate (BDP), and is designed to specifically target and treat upper and lower GI GVHD with reduced systemic immunosuppressive side effects. orBec is intended to reduce the need for systemic immunosuppressive drugs to treat GI GVHD. BDP is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP has been marketed in the US and worldwide since the early 1970s as the active pharmaceutical ingredient in a nasal spray and in a metered dose inhaler for the treatment of patients with allergic rhinitis and asthma. orBec is formulated for oral administration as a single product consisting of two tablets; one tablet is intended to release BDP in the proximal portions of the GI tract and the other tablet is intended to release BDP in the more distal portions of the GI tract.
orBec is currently the subject of an NIH supported phase II clinical trial actively enrolling patients for the prevention of GVHD as well as the subject of an NIH supported preclinical animal study in radiation injury. orBec may also have application in treating other gastrointestinal disorders characterized by severe inflammation including IBS, ulcerative colitis and Crohn's disease.
In addition to issued patents and pending worldwide patent applications held by or exclusively licensed to DOR, orBec also benefits from orphan drug designations in the US and in Europe for the treatment of GI GVHD, which provide for 7 and 10 years of post-approval market exclusivity, respectively.