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FDA approves new indication for GH therapy

Somatropin injection indicated for treatment of short stature associated with Noonan syndrome.

Issue: August 2007

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Growth hormone therapy has a new FDA-approved indication: treatment of short stature associated with Noonan syndrome.

Approximately 80% of children with Noonan syndrome are severely short statured. Somatropin injection (Norditropin, Novo Nordisk) is now the only GH therapy indicated for short stature in children with Noonan syndrome. Treatment for this disorder is typically focused on the associated clinical features and complications, such as congenital heart defects and a distinctive facial appearance, which includes hypertelorism, low-set ears and a webbed neck.

“This will increase the awareness of the diagnosis and the disorder and hopefully will encourage more practitioners to use GH therapy in patients with Noonan syndrome who are not properly growing,” Alicia Romano, MD, a pediatric endocrinologist at New York Medical College, told Endocrine Today. “This new indication will provide clinicians with more information and more confidence in prescribing it.”

Improving short stature

There are good outcome data that somatropin improved final adult height in patients with Noonan syndrome, according to Romano. Study data published in 2005 by Deborah Osio and colleagues showed that girls with the syndrome who received GH therapy for at least two years gained a mean height of 9.8 cm, and boys gained 13 cm.

“During the past few years, many clinicians have thought that GH therapy doesn’t work in patients with Noonan syndrome,” Romano said. “Some of this may be a dosing issue, and some may be that Noonan syndrome isn’t that common, and clinicians did not have a lot of experience with it.”

Noonan syndrome is rare and affects fewer than 200,000 people. 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. Somatropin has received orphan drug designation for this indication.

Children with growth failure

Along with short stature associated with Noonan syndrome, somatropin is also indicated for children with growth failure due to inadequate secretion of endogenous GH and for replacement of endogenous GH in adults with both adult- and childhood-onset GH deficiency.

Common adverse effects include rash at the injection site, lipoatrophy and headaches, glucose intolerance, fluid retention and unmasking of latent central hypothyroidism. More serious reactions may include intracranial hypertension, diabetic retinopathy and progression of preexisting scoliosis. Patients with allergy to phenol, active cancer or tumors, acute critical illness due to heart or abdomen surgery or acute respiratory failure, closed epiphyses, Prader-Willi syndrome with severe obesity or Prader-Willi syndrome with significant respiratory impairment should not use somatropin. – by Katie Kalvaitis

For more information:

• For additional information about the injection, visit the manufacturer’s website at: http://norditropin-us.com.
• Osio D, Dahlgren J, Wikland KA and Westphal O. Improved final height with long-term growth hormone treatment in Noonan syndrome. Acta Paediatr. 2005;94:1232-1237.