Friday, Oct 18, 1985

FDA Approves Genentech's Drug to Treat Children's Growth Disorder

South San Francisco, Calif. -- October 18, 1985 --

The Food and Drug Administration today approved Genentech's first human pharmaceutical, a biosynthetic growth hormone for treating severely growth retarded children.

This marks the first recombinant human pharmaceutical to be developed and marketed by a biotechnology company and only the second recombinant human pharmaceutical to be approved in the United States. The first recombinant human pharmaceutical, human insulin, approved in 1982, was also discovered by Genentech.

"With today's approval, Genentech achieves a major milestone we established at our founding -- to market our own products," Robert A. Swanson, co-founder and chief executive officer says.

The product will be sold under the brand name Protropin® (somatrem for injection). It is approved for treating children with growth hormone deficiency. An estimated 10,000 to 15,000 children in the United States suffer from growth hormone deficiency, according to the Human Growth Foundation.

Future of Recombinant DNA Technology
"Genentech's ability to produce virtually limitless quantities of therapeutically useful proteins such as human growth hormone is a clear example of the major impact that biotechnology will have on health care in the years ahead," Mr. Swanson says. "Protropin® growth hormone is the first of several products we are developing for marketing In the United States. Future products will be used to treat heart attacks, hemophilia, cancer and other life-threatening diseases." The recombinant DNA (rDNA) technology Genentech uses involves isolating the specific genetic instructions for human proteins and recombining these genes with micro-organisms. These micro-organisms act as mini-factories, producing large quantities of the desired proteins.

Replacement Therapy
Growth hormone deficiency occurs when the pituitary, a gland at the base of the brain, does not secrete adequate amounts of the protein that stimulates growth --human growth hormone (hGH). Because growth is severely retarded, afflicted children may reach an adult height of only 4 ft, looking much younger than their age during childhood and adolescence.

Until earlier this year, such children were treated with hGH extracted from the pituitary glands of human cadavers (pituitary hGH). This spring, the National Institutes of Health and two commercial suppliers halted distribution of pituitary hGH after questions were raised about possible contamination of the pituitaries with a rare and fatal virus.

"Because of the recombinant DNA process used to manufacture Protropin® growth hormone, patients will not have to worry about viral contamination associated with human tissue," says Mr. Swanson. "Thus, Protropin growth hormone will safely fill the void created when distribution of pituitary hGH was stopped."

Safe and Effective
Dr. Selna Kaplan, professor of pediatrics at the University of California, San Francisco, School of Medicine, and chief investigator for Protropin growth hormone, reports: "In tests at 13 major medical centers nationwide, Protropin® growth hormone proved to be safe and effective in stimulating growth and physical development of children with growth hormone deficiency."

Detection - Treatment
Since children with growth hormone deficiency are usually normal In size at birth, the problem often isn't identified until they enter grade school or junior high school, when the difference in size between them and their classmates becomes apparent.

The earlier treatment begins the more likely a child is to reach a more normal adult height. HGH therapy for growth hormone deficiency is only effective during the growth years, until the end of puberty when a child's linear bone growth ceases. There is no evidence that children will grow taller than their genetic potential.

Protropin® growth hormone will be prescribed primarily by pediatric endocrinologists, who are specialists in diagnosing and treating children with growth disorders. It will be available only by prescription through pharmacies at those hospitals where children suffering from growth hormone deficiency are typically treated.

In spite of the significant costs of developing Protropin®, the efficiencies of the biotechnology process will allow Protropin growth hormone to be sold by Genentech at a price per unit which is less than the previously available commercial products.

Protropin's Heritage
Genentech first produced a biosynthetic human growth hormone in 1979 and began clinical trials in 1981. To date, more than 300 children with growth disorders have been treated with Protropin growth hormone. Genentech, Inc., founded In 1976, is a leading biotechnology company with more than 850 employees involved in the development, manufacture and marketing of pharmaceuticals produced by DNA technology. Other products currently in clinical trials which Genentech plans to bring to market include Activase™ (tissue plasminogen activator) for treatment of heart attacks; gamma interferon, an anti-viral and anti-cancer agent; and TNF (tumor necrosis factor) for treating cancer patients.

# # #