PRINCETON, N.J., Aug. 4 /PRNewswire-FirstCall/ -- DOR BioPharma, Inc., (OTC Bulletin Board: DORB) (DOR or the Company), a late-stage biopharmaceutical company, announced today that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Oral BDP (beclomethasone 17,21-dipropionate, or orBec((R))) for the treatment of gastrointestinal symptoms associated with chronic Graft-versus-Host disease (cGVHD) in patients who have undergone allogeneic hematopoietic cell transplantation.
The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. In addition to providing a seven-year term of market exclusivity for orBec((R) )upon final FDA approval, orphan drug designation also positions DOR to be able to take advantage of a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees for the potential submission of a New Drug Application for orBec((R)), and certain tax credits.
orBec((R) )is a highly potent, topically active corticosteroid that has a local effect on inflamed tissue. BDP, the active component of orBec((R)), has been marketed in the United States and worldwide since the early 1970s as the active pharmaceutical ingredient in inhalation products for the treatment of patients with allergic rhinitis and asthma. orBec((R)) and oral BDP are currently in development by DOR for the treatment and prevention of GVHD, the prevention of acute radiation enteritis and the treatment of Crohn's disease.
"The FDA's decision to grant orBec((R)) Orphan Drug Designation for the treatment of GI symptoms of cGVHD marks another step in the expansion of our orBec((R))/oral BDP pipeline," stated Christopher J. Schaber, PhD, President and CEO of DOR BioPharma. "Based on data we have already generated in the treatment of acute GI GVHD, we believe that orBec((R) )has the potential to be of significant benefit to cGVHD patients."
About Chronic GVHD
Graft-versus-Host disease (GVHD) is a major complication of allogeneic hematopoietic cell transplantation. GVHD is an inflammatory disease initiated by T cells in the donor graft that recognize histocompatibility and other tissue antigens of the host, and is mediated by a variety of effector cells and inflammatory cytokines. GVHD presents in both acute and chronic forms. The symptoms of cGVHD typically present at between 100 days and three years post-transplant.
Chronic GVHD has features resembling autoimmune and other immunologic disorders such as scleroderma, Sjogren syndrome, primary biliary cirrhosis, wasting syndrome, bronchiolitis obliterans (BO), immune cytopenias and chronic immunodeficiency. The manifestations of cGVHD may be restricted to a single organ or tissue or may be widespread. Chronic GVHD can lead to debilitating consequences, e.g., joint contractures, loss of sight, end-stage lung disease, or mortality resulting from profound chronic immune suppression leading to recurrent or life-threatening infections.
Treatment of cGVHD is a challenge because it can be refractory to frontline immunosuppression. High-dose systemic corticosteroids are used with some success but carry significant toxicity.
