Novo Nordisk announced that Norditropin (somatropin [rDNA origin] injection) received US Food and Drug Administration (FDA) approval for the treatment of short stature in children with Noonan syndrome.
Noonan syndrome is defined as an autosomal dominant genetic syndrome commonly characterized by short stature, congenital heart defects, and unique facial features. The appearance of this disorder can include hypertelorism (widely-set eyes), down-slanting eyes, webbed neck, and other conditions, including congenital heart disease in half of those affected. Up to 80 per cent of children with Noonan syndrome suffer from significant short stature.
"When you see a child who has Noonan syndrome, it may not always be obvious to the naked eye, but the complications associated with the condition are quite serious and can affect both physical development and other systems," said Martin Soeters, president of Novo Nordisk Inc. "There are few treatment options available to help the physical development, and this approval marks an exciting advancement for children with this rare condition."
Noonan syndrome is classified as a rare condition with a population of less than 200,000. To encourage the development of treatments for rare disorders - that may not otherwise be commercially viable for development - the FDA designates drugs that treat fewer than 200,000 patients with an "orphan drug" designation. Norditropin has received orphan drug designation for the treatment of short stature associated with Noonan syndrome.
The prevalence of Noonan syndrome has not been determined accurately to date, but most authors report 1 in 1,000 - 2,500 live births, affecting males and females equally. Based on the United States population, prevalence may range anywhere from 125,000 to 300,000 live births. However, fetal loss can occur in Noonan syndrome so actual incidence of the disorder may be higher than its prevalence.
"Noonan syndrome is a heterogeneous genetic condition in which the clinical features are quite variable. 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," said Alicia Romano, M.D., Pediatric Endocrinologist, New York Medical College.
Norditropin (somatropin [rDNA origin] injection) is indicated for the treatment of children with short stature associated with Noonan syndrome, 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 (GHD) who meet either of the following two criteria: 1) Adult Onset: Patients who have GHD, either alone or associated with multiple hormone deficiencies (hypopituitarism), as a result of pituitary disease, hypothalamic disease, surgery, radiation therapy, or trauma; or 2) Childhood Onset: Patients who were growth hormone deficient during childhood as a result of congenital, genetic, acquired, or idiopathic causes. In general, confirmation of the diagnosis of adult GHD in both groups usually requires an appropriate growth hormone stimulation test.