Friday, Dec 21, 1990
South San Francisco, Calif. -- December 21, 1990 --Genentech, Inc. today announced that it has received permission from the Food and Drug Administration (FDA) to market Actimmune® (interferon gamma-1b) for the management of chronic granulomatous disease. It is the third pharmaceutical product developed, produced and marketed by Genentech using recombinant DNA technology, and it is the company's second product designated as an Orphan Drug by the FDA.
"Interferon gamma's approval is a testament to the true spirit of the Orphan Drug Act, which is to develop new treatments for rare diseases," said G. Kirk Raab, president and chief executive officer of Genentech. "Had it not been for the incentives provided by Congress, we could not have explored Actimmune's potential clinical application in chronic granulomatous disease patients."
Chronic granulomatous disease is a rare, inherited disorder of the immune system which affects between 250 and 400 Americans. The disease is characterized by frequent and severe infections, caused by invading bacteria and fungi, which require hospitalization and may lead to death. The only previously available treatment for this disease was frequent and high doses of antibiotics. A recently completed Phase III clinical trial showed that chronic granulomatous disease patients receiving Actimmune with antibiotic therapy had fewer and less severe serious infections and hospitalizations than chronic granulomatous disease patients receiving antibiotics alone.
"To the patients who have chronic granulomatous disease and their families who likewise suffer the stress and strain of this disease, having access to a drug that has the potential to reduce the severity of the disease's symptoms and restore them to a more normal day-to-day existence is a welcome respite," said Paul G. Quie, M.D., professor of pediatrics, Laboratory Medicine and Pathology, and Microbiology, University of Minnesota.
Interferon gamma is one of the body's proteins which is produced naturally by certain white blood cells to help reduce the frequency and severity of infections caused by invading bacteria or fungi. Since a component of the immune systems of patients with chronic granulomatous disease are dysfunctional, these patients have an impaired natural ability to defend against such infection. Based on statistical projections, interferon gamma's immune modulating activity has been shown to be effective in these patients, with an estimated 77 percent of the 63 Actimmune treated patients reported free of serious chronic granulomatous disease-related infections one year after randomization, compared with an estimated 30 percent of the 65 patients receiving placebo. These data and more will be reported in an article on the results of the study of interferon gamma in chronic granulomatous disease patients that has been submitted for publication in a national medical journal.
The FDA previously granted the company product licenses in 1985 for Protropin®, human growth hormone, as a replacement therapy for growth hormone inadequacy in children, and in 1987 for Activase®, t-PA (Alteplase), a blood clot-dissolving agent for the treatment of heart attacks. Additionally, Genentech developed human insulin, licensed to and marketed by Eli Lilly for the treatment of diabetes, and alfa interferon, licensed to and marketed by Hoffman-La Roche for the treatment of hairy cell leukemia.
Genentech, Inc. is a leading biotechnology company focusing on the development, manufacture and marketing of pharmaceuticals produced by recombinant DNA technology.
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