This approval is based on the results of a Phase 3 study conducted in Japanese subjects and a global Phase 3 clinical study, both of which were conducted in subjects with pediatric GHD, and both of which compared the efficacy and safety of once-weekly NGENLA with GENOTROPIN (somatropin), a recombinant human growth hormone for injection administered once-daily. In both studies, NGENLA showed comparable efficacy to GENOTROPIN in the primary endpoint of annual height velocity at 12 months. NGENLA 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.
‘We are pleased to receive approval for once-weekly NGENLA, which offers a new treatment option for pediatric GHD patients that can help reduce the burden associated with daily growth hormone administration. We wish to express our gratitude to the patients and their families who participated in the clinical studies and to all the sites conducting these trials,’ said
Product name: NGENLA Inj.60mg Pens
General name: Somatrogon (recombination)
INDICATIONS: Short stature due to growth hormone deficiency without closed epiphyses
DOSAGE AND ADMINISTRATION
Generally, Somatrogon (recombination) 0.66 mg per kilogram body weight is administered once-weekly by subcutaneous injection.
Marketing Authorization Holder
About the Japan Phase 3 Study
The Phase 3 study of NGENLA in 44 treatment-naive 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 NGENLA 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 NGENLA 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, NGENLA 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.
About the Global Phase 3 Study
The Global Phase 3 study of NGENLA in 224 treatment-naive children with pediatric GHD in over 20 countries was a 12-month randomized, open-label, active-controlled study evaluating the safety and efficacy of weekly NGENLA (somatrogon) injection compared to GENOTROPIN (somatropin) administered once-weekly. Eligible patients 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.
About NGENLA (somatrogon) injection
NGENLA is a 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. NGENLA was approved in
GENOTROPIN (somatropin) is a man-made, prescription treatment option, approved in
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 people1,2. In children, this disease can be caused by genetic mutations or acquired after birth1,3. 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 delayed1,3,4. Children may also experience other problems with physical health and mental well-being3,4.
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The information contained in this release is as of
This release contains forward-looking information about a product candidate, NGENLA (somatrogon), a once-weekly long-acting recombinant human growth hormone, including an approval by the
A further description of risks and uncertainties can be found in
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2: Stanley T. Diagnosis of growth hormone deficiency in childhood. Curr Opin Endocrinol Diabetes Obes. 2012;19(1):47-52. doi:10.1097/MED.0b13e32834ec952.
3: Cerbone M, Dattani MT. Progression from isolated growth hormone deficiency to combined pituitary hormone deficiency. Growth Horm IGF Res. 2017;37:19-25. doi:10.1016/j.ghir.2017.10.005.