DOR BioPharma's orBec(R) Receives Orphan Drug Designation in Australia for the Treatment of GI GVHD
Tuesday November 25, 8:13 am ET
EWING, NJ--(MARKET WIRE)--Nov 25, 2008 -- DOR BioPharma, Inc. (DOR or the Company) (OTC BB:DORB.OB - News), a late-stage biopharmaceutical company developing products to treat the life-threatening side effects of cancer treatments and serious gastrointestinal diseases, and vaccines against certain bioterrorism agents, announced today that the Therapeutics Goods Administration (TGA) of Australia has designated orBec® as an Orphan Drug for the treatment of patients with gastrointestinal Graft-Versus-Host-Disease (GI GVHD) following allogeneic hematopoietic cell transplantation.
The Australian Orphan Drug Program was launched in 1998 by the TGA and is intended to encourage sponsors to market orphan drugs in Australia to ensure the availability of a greater range of treatments for rare diseases (those with a prevalence of 2,000 or fewer patients/subjects annually in the Australian population). Once orphan designation is granted, the TGA waives evaluation fees and sets up a priority evaluation pathway for processing orphan drugs.
"The TGA's decision to grant Orphan Designation to orBec® is extremely exciting and validates the potential of this therapy to provide a therapeutic option to patients who are afflicted with GI GVHD," said John Michailidis, General Manager of Orphan Australia. "We look forward to working with DOR to make this product available within Australia, as well as in New Zealand and South Africa."
"We are pleased with the TGA's decision to grant orBec® Orphan Drug Designation for the treatment of GI GVHD," stated Christopher J. Schaber, PhD, President and CEO of DOR BioPharma. "This bolsters our intellectual property position in Australia, which already includes issued and pending patents covering orBec® in GI GVHD. It also potentially provides for a quicker path to regulatory approval in Australia once we have successfully completed our confirmatory Phase 3 clinical trial of orBec®."
Dr. Schaber added, "We have also launched our orBec® Named Patient Access Program in Australia and have booked our first sales pursuant to this important program. We look forward to a prosperous relationship with Orphan Australia Pty Ltd, and with their assistance hope to help as many GI GVHD patients in Australia as possible."
About orBec®
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 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 distal portions of the GI tract.
Two prior randomized, double-blinded, placebo-controlled Phase 2 and 3 clinical trials demonstrated that orBec® provides clinically meaningful outcomes when compared with the current standard of care, including a lowered exposure to systemic corticosteroids following allogeneic transplantation. Currently, there are no approved products to treat GI GVHD. The Phase 3 trial was a 129-patient pivotal Phase 3 multi-center clinical trial of orBec® conducted at 16 leading bone marrow/stem cell transplantation centers in the US and France. Although orBec® did not achieve statistical significance in the primary endpoint of its pivotal trial, namely median time to treatment failure through Day 50 (p-value 0.1177), orBec® did achieve statistical significance in other key secondary endpoints such as the proportion of patients free of GVHD at Day 50 (p-value 0.05) and Day 80 (p-value 0.005) and the median time to treatment failure through Day 80 (p-value 0.0226), as well as a 66% reduction in mortality among patients randomized to orBec® at 200 days post-transplant with only 5 patient (8%) deaths in the orBec® group compared to 16 patient (24%) deaths in the placebo group (p-value 0.0139). At one year post randomization in the pivotal Phase 3 trial, 18 patients (29%) in the orBec® group and 28 patients (42%) in the placebo group died within one year of randomization (46% reduction in mortality, hazard ratio 0.54, 95% CI: 0.30, 0.99, p=0.04, stratified log-rank test).
About GI GVHD
GI GVHD is a debilitating and painful disease. It is a common disorder among immuno-compromised cancer patients after receiving allogeneic hematopoietic cell transplants. Unlike organ transplants where the patient's body may reject the organ, in GVHD it is the donor cells that begin to attack the patient's body -- most frequently the gastrointestinal tract, liver and skin. Patients with mild-to-moderate GI GVHD typically develop symptoms of anorexia, nausea, vomiting and diarrhea. If left untreated, GI GVHD can progress to ulcerations in the lining of the GI tract, and in its most severe form, can be fatal. |
