Top-line results show safety, efficacy for long-acting prepubertal GH
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Opko Health and Pfizer announced positive top-line results this week for an investigational long-acting growth hormone injection, noting the therapy was safe and effective for prepubertal children with GH deficiency, according to a press release from the two companies.
The companies stated that once-weekly somatrogon injection met its primary endpoint of noninferiority to daily somatropin injection (Genotropin, Novo Nordisk), as measured by annual height velocity at 12 months.
“We are very pleased to announce these favorable top-line results demonstrating the efficacy of somatrogon dosed once-weekly in the pediatric patient population with GH deficiency,” Phillip Frost, MD, chairman and CEO of Opko, said in the release. “Somatrogon is a new molecular entity incorporating Opko’s proprietary long-acting technology. We believe somatrogon represents a significant advance in the treatment of children with GH deficiency compared to the current standard of one injection per day that could enhance a patient’s adherence to treatment and quality of life.”
Researchers found that once-weekly somatrogon was noninferior to once-daily somatropin with respect to height velocity at 12 months, with the least square mean higher in the somatrogon group vs. the somatropin group (10.12 cm/year vs. 9.78 cm/year). The treatment difference in height velocity was 0.33 cm per year (95% CI, –0.39 to 1.05), according to the release.
Change in height standard deviation scores at 6 and 12 months, key secondary endpoints, were higher in the somatrogon group vs. the somatropin group. At 6 months, change in height velocity was similarly higher for children treated with somatrogon vs. children who received somatropin.
Somatrogon was generally well tolerated in the study and comparable to that of somatropin dosed once daily with respect to the types, numbers and severity of adverse events observed between the treatment arms. The companies stated that immunogenicity testing and additional data analysis are ongoing.
“We’re encouraged by these data and look forward to the possibility of bringing this longer-acting therapy to children,” Brenda Cooperstone, MD, chief development officer of rare disease at Pfizer Global Product Development, said in the release. “If approved, somatrogon could reduce the daily disease burden on children and their caregivers, potentially increasing treatment adherence.”
In 2014, Pfizer and Opko entered into a worldwide agreement for the development and commercialization of somatrogon for the treatment of GH deficiency. Under the agreement, Opko is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing the product.
The full results of the study will be submitted for presentation at a future scientific meeting. – by Regina Schaffer
Disclosures: Cooperstone is chief development officer of rare disease at Pfizer Global Product Development. Frost is chairman and CEO of Opko.