Somavaratan effective for improving growth rates in children with GH deficiency

ORLANDO, Fla. — A twice-monthly investigational therapy for children with growth hormone deficiency was effective for increasing growth rates when the dose was increased to 3.5 mg/kg twice per month, according to study findings presented here.

“We all know that rhGH for treatment of pediatric GH deficiency has been established as a daily therapy and the primary treatment for GH deficiency for the past 3 decades,” Bradley Scott Miller, MD, PhD, associate professor, division of pediatric endocrinology at the University of Minnesota Masonic Children’s Hospital, said during his presentation. “The current challenges include that this requires daily injections subcutaneously, and that requires compliance to get the best outcomes. In reports of compliance, we can see that as many as 77% of children and adults are noncompliant with their GH deficiency treatment.”

Miller and colleagues evaluated 48 prepubertal children (mean baseline age, 7.5 years) with GH deficiency treated with the recombinant human GH, or rhGH, somavaratan (Versartis Inc.). The children had previously participated in the VERTICAL (0-6 months) and VISTA (6-12 months) trials to determine 3-year long-term outcomes with somavaratan.

Researchers transitioned all participants to a uniform frequency and dose of 3.5 mg/kg twice per month by the beginning of year 2 based on early growth and insulin-like growth factor I responses to treatment.

Through 3 years of treatment, mean height velocity remained consistent and height standard deviation scores increased from -2.6 at baseline to -1.9 at 1 year, -1.4 at 2 years and -1 at 3 years. At year 3, the mean difference in years between bone age and chronological age improved to -0.6 from -1.53 at baseline.

All reported adverse events were mild to moderate.

“The continued long-term follow-up and data from patients on somavaratan therapy is important to help inform and build confidence in the pediatric endocrine community when considering somavaratan as a pediatric treatment option if it receives future FDA approval,” study researcher Patricia Y. Fechner, MD, associate professor of pediatric endocrinology at the University of Washington and Seattle Children’s Hospital, told Endocrine Today. “More robust data on patients switching from daily rhGH to somavaratan would also be desirable as the majority of our patients are currently on daily therapy already, and this is understudied. If approved, a phase 4 study would take place to further provide data to physicians on dose titration.” – by Amber Cox

Reference:

Moore WV, et al. OR31-1. Presented at: The Endocrine Society Annual Meeting; April 1-4, 2017; Orlando, Fla.

Disclosures: Fechner reports financial ties with Versartis. Miller reports various financial ties with Abbvie, Alexion, Armagen, BioMarin, Eli Lilly, Endo Pharmaceuticals, Ferring Pharmaceuticals, Genentech Inc., Novo Nordisk, Pfizer Inc., Sandoz, Shire, Tolmar, Up to Date and Versartis.