It appears a main area of focus among biopharmaceutical companies currently is having a treatment for growth hormone deficiency that reduces or eliminates need for daily injections. Novo Nordisk was one of those who presented clinical data at the 2018 annual meeting of the European Society for Pediatric Endocrinologist on a drug that can potentially function as a once-a-week treatment for people suffering from deficiency issues.
The global healthcare company presented results from its REAL 3 trial, showing somapacitan’s potential to function satisfactorily as a once-weekly treatment for childhood growth hormone deficiency.
The condition, which is also known as pediatric growth hormone deficiency (PGHD), is a rare disorder. It is characterized by low circulating growth hormone. This hinders normal growth and results in short stature in children.
At the moment, there are no approved treatments for this condition that can be used only once a week. Treatment typically requires daily injections.
An alternative treatment
Somapacitan is one of the substances currently being developed to reduce reliance on daily injections. It is a long-acting derivative of human growth hormone (HGH).
It has been modified to have greater binding capacity to the plasma protein albumin. This makes it potentially helpful as a treatment that needs to be administered just once a week.
The clinical trial in focus compared somapacitan to the once-a-day Norditropin by the same company for children with growth hormone deficiency.
The long-acting growth hormone delivered similar benefits as the FDA-approved medication.
About the trial
The REAL 3 trial was a randomized, active-controlled, multinational trial. The researchers studied the effect in 59 pre-pubertal children suffering from growth hormone deficiency. Their aim was to find out how multiple doses of the long-acting substance compared to daily use of Norditropin.
At random, the children in the study were given any of three doses of somapacitan (0.04 mg/kg, 0.08 mg/kg, or 0.16 mg/kg per week) or 0.034 mg/kg of Norditropin per day. This continued for a period of about 26 weeks.
How well each of the doses of the persistent growth hormone compared to the once-daily treatment depended on the size.
The 0.04 mg/kg and 0.08 mg/kg doses led to 8.0 cm and 10.9 cm mean annualized height velocity respectively. The 0.16 mg/kg dose resulted in 12.9cm improvement. By comparison, the mean annualized height velocity for the Norditropin group was 11.4 cm.
Difference in height velocity between the recombinant HGH and somapacitan at the latter’s two higher doses wasn’t significant.
Relief for children with growth hormone deficiency
The encouraging Phase 2 clinical data means patients with childhood growth hormone deficiency may soon have a better treatment option.
The current requirement for daily injections is difficult to administer or stick to, considering the discomfort to children.
“Today children with growth hormone deficiency require daily injections, which can be a considerable treatment burden for patients and caregivers,” lead researcher Professor Lars Savendahl said. “A once-weekly treatment would represent a significant development for these children and their families, helping them to live less disrupted lives.”
The safety profile of somapacitan as seen in the trial was similar to that of Norditropin.
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