OPKO Health has completed enrolment in a global pivotal Phase lll trial of somatrogon (hGH-CTP) for the treatment of growth hormone deficient (GHD).
The randomised, open-label, active-controlled trial enrolled around 225 treatment-naïve children with growth hormone deficiency (GHD).
Currently being conducted in more than 30 countries worldwide, the trial is expected to investigate a single weekly injection of somatrogon, which will be delivered via a multi-dose disposable pen.
The enrolled patients were randomised in 1:1 ratio into two arms, one of which will receive once-weekly somatrogon and the other will receive a once-daily dose of Genotropin.
The trial’s primary endpoint is height velocity at 52 weeks, while secondary endpoints are safety and pharmacodynamic endpoints.
Children completing the Phase lll trial may get a chance to enrol in an open-label long-term extension, where they will receive somatrogon.
OPKO Health Chairman and CEO Phillip Frost said: “We are pleased to have completed enrolment for this study, as the pediatric segment represents approximately 80% of the market for the treatment of GHD.
“We are hopeful that the outcome of this study will support a dosing change from daily to weekly administration and positively impact the quality of life for children with GHD.”
Somatrogon (hGH-CTP) is a new molecular solution designed to maintain the natural sequence of growth hormone, fused with a C-terminus peptide to extend its half-life.
In a Phase ll trial where GHD children were treated with once-weekly injections of somatrogon, it reportedly produced pharmacodynamic effects similar to daily growth hormone replacement therapy with comparable efficacy and safety.
Somatrogon has already received orphan drug designation in the US and the European Union (EU) for use in the treatment of children and adults with GHD.