This submission is based on the results of the Japan Phase 3 and global Phase 3 clinical studies, conducted in subjects with pediatric GHD, in which the efficacy and safety of somatrogon administered once weekly were compared with GENOTROPIN® (somatropin), a recombinant human growth hormone for injection, administered once daily. In both studies, somatrogon showed comparable efficacy to GENOTROPIN for the primary endpoint of annual height velocity at 12 months of treatment. Somatrogon was generally well tolerated in both studies, with comparable safety to that of GENOTROPIN administered once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms.
In 2014, Pfizer Inc. and
About the Japan Phase 3 Study
The Phase 3 study of somatrogon in 44 treatment-naïve Japanese pre-pubertal children with pediatric GHD was a 12-month, open-label, randomized, active-controlled, parallel-group study of the efficacy and safety of weekly somatrogon compared to recombinant human growth hormone (r-hGH), GENOTROPIN® (somatropin) for injection treatment administered once-daily. Eligible patients were randomized in a 1:1 ratio to receive either once-weekly somatrogon or GENOTROPIN administered once-daily (reference therapy, 0.025 mg/kg/day which is equivalent to 0.175 mg/kg/week). To obtain pharmacokinetic information of three different weekly doses in Japanese pediatric GHD patients, somatrogon treated patients received 0.25 mg/kg/week for 2 weeks, followed by 0.48 mg/kg/week for 2 weeks followed by 0.66 mg/kg/week for the remaining 46 weeks. Somatrogon was administered subcutaneously using a single patient use, multi-dose, disposable, pre-filled pen, the same pen used in the global study, while GENOTROPIN was administered using approved commercial products in
About Somatrogon
Somatrogon is an investigational biologic product that is glycosylated and comprises the amino acid sequence of human growth hormone and one copy of the C-terminal peptide (CTP) from the beta chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies of CTP (in tandem) at the C-terminus. The glycosylation and CTP domains account for the half-life of the molecule. Somatrogon has received Orphan Drug designation in the
About Growth Hormone Deficiency
Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland and affects one in approximately 4,000 to 10,000 people. In children, this disease can be caused by genetic mutations or acquired after birth. Because the patient's pituitary gland secretes inadequate levels of somatropin, the hormone that causes growth, his or her height may be affected and puberty may be delayed. Without treatment, he or she will have persistent growth attenuation, a very short height in adulthood, and may experience other health problems.
About GENOTROPIN
GENOTROPIN (somatropin) is a man-made, prescription treatment option, approved in
About
OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit http://www.OPKO.com.
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