A new drug approved for treating severe short stature is in trials for less severe growth conditions.

The drug, Increlex, was approved by the FDA on August 31 for the most severe form of short stature due to a deficiency of insulin-like growth factor-1 (IGF-1).

Researchers are now testing the drug to see if it can be used for less severe growth disease.

“For decades, the only available drug treatment for short stature has been shots of growth hormone,” says Richard Levy, a pediatric endocrinologist at Rush University Medical Center. “However, patients who are deficient in IGF-1 and resistant to the effects of growth hormone do not respond well, if at all, to the shots.”

Instead of using growth hormone to stimulate the production of IGF-1, says Levy, the goal is to replace the IGF-1 directly.

A Rush news release reports that Increlex is a genetically engineered copy of IGF-1 injected daily before a meal to provide the catalyst the body needs to grow.

The release states that the FDA’s approval of Increlex is based on clinical trial data from 71 patients.

The trial showed a statistically significant increase in growth rate over an eight-year period in response to Increlex therapy, with children gaining an additional inch per year for each year of therapy over the course of eight years.

The drug also appears to be well-tolerated.

More here.