New Treatment for Noonan Syndrome

FDA OK's DRUG FOR NOONAN SYNDROME

June 1, 2007

WASHINGTON, June 1 (UPI) -- The U.S. Food and Drug Administration Friday approved Copenhagen-based Novo Nordisk's Norditropin for children with Noonan syndrome.

The genetic disorder is characterized by short stature, congenital heart defects and unique facial features, notably widely set eyes.

The use of Norditropin -- somatropin injection -- is aimed at treatment of short stature in these individuals.

Fewer than 200,000 people have the condition, prompting the FDA to give Norditropin an orphan drug designation. No other treatment for short stature in these patients is available.

The prevalence of Noonan syndrome has not been determined accurately to date, but most authors report 1 in 1,000 to 1 in 2,500 live births, affecting males and females equally. In addition to heart abnormalities and short stature -- affecting 80 percent of people with Noonan syndrome -- patients may also have visual and hearing deficits, bleeding abnormalities and lack muscle tone.

"Noonan syndrome is a heterogeneous genetic condition in which the clinical features are quite variable," said Alicia Romano, a pediatric endocrinologist at the New York Medical College, in a statement issued by Novo Nordisk.

"Short stature, which can be severe, is one of the most common characteristics. Treatment with Norditropin may help children with Noonan syndrome improve one of the most concerning physical features of the condition."

Norditropin is also approved for treatment of children with growth failure due to inadequate secretion of endogenous growth hormone and for replacement of endogenous growth hormone in adults with growth hormone deficiency.

United Press International