Monday, Oct 25, 1993

FDA Advisory Committee Unanimously Recommends Approval for Nutropin as Treatment for Growth Failure in Children with Chronic Renal Insufficiency

South San Francisco, Calif. -- October 25, 1993 --

The Endocrinologic and Metabolic Drugs Advisory Committee of the Food and Drug Administration (FDA) today recommended that the FDA grant marketing approval for Genentech's new recombinant human growth hormone Nutropin® (somatropin for injection) as a treatment for growth failure in children due to chronic renal insufficiency (CRI) before transplantation. The vote of the independent panel of experts was unanimous (8-0) in favor of approving Nutropin.

CRI occurs in children with irreversible damage to the kidneys and causes a variety of medical problems in children, including growth failure. With the advent of kidney transplants and the availability of dialysis, children with CRI now reach adulthood, but many still experience growth problems. The condition affects an estimated 3,000 children in the United States. Nutropin has been designated an orphan drug for the treatment of growth failure in children with CRI.

"We're particularly pleased with the Advisory Committee's recommendation, because it takes us one step closer to providing another product that meets a significant unmet medical need," said G. Kirk Raab, Genentech's president and chief executive officer. "We will continue to work closely with the FDA to gain final approval for Nutropin so it can be made available as quickly as possible to the children who need it."

Clinical Trial Results

Results of ongoing Phase III clinical trials with 195 patients show Nutropin therapy administered prior to renal transplantation significantly increases growth velocity and reduces height deficit. The data suggest that after transplantation, patients maintain height gains achieved with Nutropin therapy. Normal advancement of bone age occurs in these patients, suggesting that height gains will result in a greater final height.

Height in the normal range was achieved in 65 percent of the patients treated with Nutropin for two years, and 91 percent of patients treated for at least three years.

The safety profile of Nutropin during the studies did not differ substantially from that reported for growth hormone treatment of patients with growth hormone inadequacy. Progression of renal disease was not altered by Nutropin therapy. Genentech will continue to monitor the long-term efficacy and safety of Nutropin in Phase IV post-marketing surveillance studies.

The goal of administering growth hormone to children with CRI is to improve growth before renal transplantation is performed. Renal transplantation may help a child start growing normally again, but most of these children do not have "catch-up" growth to offset the loss of growth due to CRI.

Before the use of Nutropin, no interventions proved successful at correcting the severe height deficit these children experience. If approved, Nutropin will be the first and only effective treatment to address growth failure children with CRI.

"We've tried a number of methods over the years, such as nutritional supplementation, but we were not able to improve growth," said Edward Kohaut, M.D., professor of pediatrics, director of nephrology at the University of Alabama, Birmingham and one of the principal clinical investigators. "For the first time we have a potentially beneficial way to help these children grow..

Nutropin is a new natural sequence human growth hormone (hGH) produced by Genentech using recombinant DNA technology. Nutropin mimics the activity of hGH produced in the human body.

Genentech, Inc. is a leading biotechnology company that discovers, develops, manufactures and markets human pharmaceuticals for significant medical needs. The company has headquarters in South San Francisco, Calif., and is traded on the New York and Pacific Stock Exchanges under the symbol GNE.

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