Accentia Biopharmaceuticals, Inc. and its majority-owned subsidiary, Biovest International, Inc. jointly announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Revimmune, the company’s proprietary system-of-care based on high-dose administration of Cytoxan (cyclophosphamide), for the prevention of Graft-Versus-Host Disease (GVHD) following bone marrow transplant.

GVHD is a life-threatening autoimmune complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted cells trigger an immune attack on the transplant recipient's body.

With FDA Orphan Drug Status, Accentia gains seven years of market exclusivity for Revimmune for the prevention GVHD following bone marrow transplant upon its approval by the FDA, thereby offering competitive protection from similar drugs of the same class. Orphan Drug Status also provides Accentia with eligibility to receive potential tax credit benefits, potential grant funding for research and development and significantly reduces the requisite filing fees for marketing applications.

US Congressman C W “Bill” Young (Florida, 10th District) commented on the milestone, “Bone marrow transplantation offers the gift of life to thousands of men, women and children with otherwise fatal blood disorders, and I support everything we can do to give these patients the best chance at success. Graft-versus-host is a serious complication in many cases, and I am pleased that Accentia Biopharmaceuticals is addressing this very issue and with its new therapy may improve the success rate of many bone marrow transplants. With each and every bone marrow transplant that is made possible through the national registry, we learn something new that helps all future patients achieve a better outcome.” The C W Bill Young Department of Defence (DoD) Bone Marrow Donor Program was named for Congressman Young, who initiated and supported the development of the National Marrow Donor Program and the DoD program for unrelated donor marrow transplantation.

Dr Carlos F Santos, PhD, Accentia’s chief scientific officer, commented, “Two phase I/II clinical trials conducted by physicians at Johns Hopkins University have provided important data regarding the safety and efficacy of Revimmune for prevention of GVHD following partially matched bone marrow transplants. We consider these outcomes together with data from an additional multi-centre study to be highly encouraging. We are now preparing for meetings with regulatory agencies, including the FDA, to discuss the next steps for the regulatory approval of Revimmune for the prevention of GVHD following bone marrow transplantation, in particular those from partially-matched donors.”

The Company holds an exclusive world-wide license to Revimmune from Johns Hopkins University and has a strategic agreement with Baxter Healthcare Corporation covering the commercialization of Cytoxan, the active ingredient of Revimmune, for the treatment of a range of autoimmune diseases including multiple sclerosis and the prevention of graft-versus-host disease following bone marrow transplant.

The Department of Defence established the C W Bill Young Department of Defence Marrow Donor Centre in Washington, DC to support DoD volunteer marrow donors, and the Centre coordinates all the medical and logistic support for DoD personnel who volunteer for the possibility of donating marrow.

Accentia holds the worldwide exclusive license to Revimmune for the treatment of autoimmune diseases, such as multiple sclerosis. Developed by Dr Richard Jones, Dr Robert Brodsky, and colleagues at Johns Hopkins University School of Medicine, Revimmune uses an already-approved active pharmaceutical (Cytoxan) in a novel, patent-pending system-of-care capable of "rebooting" a patient's immune system. Revimmune therapy is believed to act by completely eliminating mature lymphocytes throughout the body while selectively sparing immune stem cells in the bone marrow. Shortly following a course of Revimmune, a natural "rebooting" process takes place as bone marrow stem cells repopulate the immune system with healthy immune cells that no longer possess the traits of autoimmunity.

A strategic agreement with Baxter Healthcare Corporation provides Accentia with the exclusive, worldwide right to purchase Baxter’s cyclophosphamide, which is marketed under the brand name Cytoxan, for the treatment of designated autoimmune diseases including multiple sclerosis.

Accentia Biopharmaceuticals, Inc. is committed to advancing the autoimmune disease therapy, Revimmune, as a comprehensive system of care and drug regimen designed for the treatment of autoimmune diseases.