hGH-CTP is a once-weekly injectable, created using recombinant technology, for the treatment of pediatric and adult growth hormone deficiencies. The molecule consists of the natural peptide sequence of native growth hormone and the 28 amino acids of the C-Terminus Peptide of the human chorionic gonadotropin hormone. This molecule, as compared to current GH replacement therapies, is designed to reduce the injection frequency from a daily to once a week in adults and children with GH deficiencies.
Adult growth hormone deficiency (AGHD) afflicts approximately 50,000 people in the U.S. There are two main causes: damage or trauma to the pituitary or hypothalamus gland, and being born with a growth hormone deficiency. Whereas growth hormone is principally used to assist growth in children, growth hormone is necessary for human growth hormone (hGH) deficient adults to maintain muscle, bone and fat tissues in a healthy balance and quality of life. With this deficiency, fat is deposited more easily around the middle of the abdomen; there's a decrease in muscle; bones become weaker resulting in osteoporosis; and cholesterol levels become higher, with a decrease in the "good" HDL cholesterol. hGH is also used in adults with AIDS-induced weight loss. Currently, patients receiving hGH must have daily injections administered six or seven times a week.
Growth hormone deficiency (GHD) in children is defined as growth failure associated with inadequate growth hormone production. Approximately 80% of the more than $3 billion market for hGH is for Pediatric growth hormone deficiency. Current treatment of pediatric GHD patients is done using daily injections of recombinant GH.
OPKO Health's hGH-CTP is a once a weekly injection that utilizes CTP technology to enhance the half-life and maintain the natural native sequence of growth hormone. A long term phase 2 study of hGH-CTP, in pediatric GHD patients, has been on-going since 2013. Over 80% of the study patients continue with hGH-CTP treatment.
In December 2016, OPKO commenced a pivotal phase 3 clinical trial of hGH-CTP to treat pediatric GHD patients. The enrollment of this phase 3 global study was completed in August 2018, enrolling 228 enrolled patients in 21 countries. The study is designed as a non-inferiority comparison between weekly administration of hGH-CTP and daily injections of Genotropin. Both treatments are administered via a ready to use, multiple dose, disposable, refrigerated pen injection device. In parallel, a phase 2/3 product registration clinical study in pediatric Japanese GHD patients has completed enrollment. This study is designed to evaluate the pharmacokinetic profile of hGH-CTP in Japanese pediatric GHD patients and compare the efficacy and safety between weekly hGH-CTP and daily Genotropin.