The target Prescription Drug User Fee Act (PDUFA) action date for decision by the FDA is in
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The submission is supported by the results of a global Phase 3 trial evaluating the safety and efficacy of somatrogon administered once weekly to pediatric patients with GHD. This study met its primary endpoint of non-inferiority compared to GENOTROPIN (somatropin) for injection administered once daily, as measured by annual height velocity at 12 months. The top-line results from the study demonstrated the least square mean was higher in the somatrogon group (10.12 cm/year) than in the somatropin group (9.78 cm/year); the treatment difference (somatrogon - somatropin) in height velocity (cm/year) was 0.33 with a two-sided 95% confidence interval of the difference (-0.39, 1.05). In addition, change in height standard deviation scores at 6 and 12 months, key secondary endpoints, were higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort. Moreover, at 6 months, change in height velocity, another key secondary endpoint, was higher in the somatrogon dosed once-weekly cohort in comparison to the somatropin dosed once-daily cohort. These common measures of growth are employed in the clinical setting to measure the potential level of catch-up growth that subjects may experience relative to the heights of their age and gender matched peers.
Somatrogon was generally well tolerated in the study and comparable to that of somatropin administered once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms.
In 2014,
ABOUT THE STUDY
The somatrogon Phase 3 trial is a randomized, open-label, active-controlled study conducted in over 20 countries. This study enrolled and treated 224 pediatric patients, treatment-naive children with growth hormone deficiency who were randomized 1:1 into two arms: somatrogon administered at a dose of 0.66 mg/kg body weight once-weekly vs GENOTROPIN (somatropin) administered at a dose of 0.034 mg/kg body weight once daily. The primary endpoint of the trial was height velocity at 12 months. Secondary endpoints included change in height standard deviation at 6 and 12 months, safety and pharmacodynamic measures. Children completing this study had the opportunity to enroll in a global, open-label, multicenter, long-term extension study, in which they were able to either continue receiving or switch to somatrogon. Approximately 95% of the patients switched into the open-label extension study and received somatrogon treatment.
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 (SOMATROPIN)
GENOTROPIN is a man-made, prescription treatment option, approved in
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