Human Genome Sciences Receives Orphan Drug Designation for BLyS Therapeutic Protein for Treatment of Common Variable Immunodeficiency
One of Four HGS Drugs in Clinical Trials
ROCKVILLE, Md., Feb. 27 /PRNewswire/ -- Human Genome Sciences, Inc. (Nasdaq: HGSI - news) today announced that BLyS(TM), one of its four drugs in human clinical trials, has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its first indication, the treatment of common variable immunodeficiency (CVID).
Orphan drug designation entitles Human Genome Sciences to exclusive marketing rights in the United States on BLyS for seven years following marketing approval and enables the company to apply for research funding, tax credits for certain research expenses and a waiver from the FDA's application user fee. In the past, orphan drug designation has been associated with other biopharmaceuticals such as Avonex for multiple sclerosis. Other biopharmaceuticals that have been indicated for orphan drug status include Epogen and Neupogen.
Although CVID is the first and only indication for which Human Genome Sciences has sought orphan drug status, it is by no means the only disease for which BLyS may be appropriate. The discovery of BLyS protein may lead to therapies for several diseases that involve B cells, including immune deficiencies, autoimmune disease and B cell tumors. Human Genome Sciences' drug development teams are advancing several therapeutic concepts based on the discovery of BLyS, including not only the BLyS therapeutic protein but also BLyS monoclonal antibody and radiolabeled BLyS.
Sally D. Bolmer, Vice President, Regulatory, said, ``The FDA's Orphan Drug Program provides a number of benefits to Human Genome Sciences. BLyS and CVID are a very good example of why the orphan drug program was created. We are very pleased to be able to take advantage of this innovative FDA program.''
Common Variable Immunodeficiency, or CVID, is one of a group of primary immunodeficiency disorders that are characterized by increased susceptibility to infection. Affected individuals suffer from multiple bouts of pneumonia, bronchitis and sinusitis as well as other infections. Patients suffering from CVID require life-long treatment with antibodies to protect against recurrent infection.
BLyS is a protein made by the human body that stimulates the production of antibodies. Antibodies recognize foreign substances and help defend the body against infection by viruses, bacteria and other microorganisms. Patients with CVID fail to make normal quantities of antibodies, or immunoglobulins, making them susceptible to infections. The goal is that by providing BLyS to these patients, they will be able to produce their own antibodies and ward off infections themselves. BLyS is currently in an on-going Phase I clinical study.
Arthur M. Mandell, Senior Vice President and Chief Business Officer, said, ``Human Genome Sciences intends to be a fully integrated biopharmaceutical company with its own marketing and own sales force to promote its products to physicians. The characteristics of the CVID market provide an opportunity for us to establish a commercial infrastructure that could be expanded as additional products reach the market.''
Craig A. Rosen, Executive Vice President, Research and Development, said, ``We hope that BLyS will become an important treatment opportunity for CVID patients. The discovery of BLyS has yielded many unique product opportunities for Human Genome Sciences. In addition to BLyS therapeutic protein, these include BLyS monoclonal antibody, to neutralize BLyS protein, which may address the needs of patients with systemic lupus erythematosus (SLE) (1) and radiolabeled BLyS which may be used to treat patients with B cell cancers such as multiple myeloma. (2) ''
William A. Haseltine, Ph.D., Chairman and Chief Executive Officer, said, ``We are pleased to receive this orphan drug designation for BLyS for CVID; orphan drug designation can facilitate the development of drugs for certain types of diseases. Receipt of the orphan drug status for BLyS is another step in HGS' development as a fully-integrated biopharmaceutical company that discovers, develops, manufactures and eventually sells our own drugs.''
Human Genome Sciences is a company with the mission to treat and cure disease by bringing new gene-based drugs to patients. |
