Pfizer Announces Top-Line Results From Phase 3B Study Of GENOTROPIN

Pfizer Inc. announced positive top-line results from a Phase IIIB study assessing GENOTROPIN (somatropin) in infants. GENOTROPIN is a prescription drug intended for the treatment of growth failure in pediatric patients.

The Phase 3B study assessed GENOTROPIN’s (somatropin) 24-month efficacy on the height in small-for-gestational-age (SGA) children 24-30 months old. The study met its primary endpoint of statistically significant differences at 24 months in change from baseline height between patients treated with GENOTROPIN and those in the untreated control group, as measured by height standard deviation score (SDS).

GENOTROPIN is a man-made, prescription treatment option by Pfizer for the treatment of growth failure in children. The drug is approved in the United States for the following conditions: children who are unable to produce sufficient growth hormone on their own, children who have the genetic condition called Prader-Willi syndrome (PWS), children who have the genetic condition called Turner syndrome (TS) or have idiopathic short stature (ISS), and children who were born smaller than average. GENOTROPIN is also approved for the treatment of adults with growth hormone deficiency. The drug is similar to the natural growth hormone that our bodies make and has a proven safety profile.

Growth failure can be caused by growth hormone deficiency (GHD) and idiopathic ISS, among others. GHD means the patient’s body does not produce enough growth hormone on its own. ISS indicates that patients are shorter than 98.8% of other children of the same age and sex. Children with ISS grow at a rate that is unlikely to allow them to reach normal adult height. Growth plates of children with ISS have not closed. ISS has no known cause or origin.

Thirty-nine patients completed the multi-center, controlled study which randomized SGA children to Genotropin or an untreated control group. Forty-three participants between 24-30 months old from 16 centers in eight European countries including Czech Republic, Germany, Sweden, Spain, Italy, Belgium, Switzerland, and Netherlands participated in the study.