Pfizer's new drug for childhood growth hormone deficiency, somatrogon, is under review

GHD in children is a serious and rare disease caused by insufficient growth hormone secretion in the pituitary gland.
children with GHD are not only short in stature, but also have metabolic abnormalities, psychosocial challenges, cognitive impairment and poor quality of life.
, GHD's standard of care has been to inject human growth hormone (hGH) once a day subskin to improve growth and metabolic effects.
for caregivers and patients, the burden of daily injections is high, which can lead to poor compliance and reduce overall treatment effectiveness.
somatrogon is a new molecular entity that contains a natural sequence of human growth hormone and contains a copy at the N end and a human fluffy membrane gonot hormone (hCG) β chain C-end peptide (CTP) at the N end, which prolongs the half-life of the molecule.
the United States and the European Union, somatrogon has been granted the orphan drug qualification (ODD) for the treatment of GHD children and adults.
studies to date have shown that weekly somatrogon significantly reduces lifestyle disturbances, supports patient preferences, and improves compliance compared to hGH once a day.
2014, Pfizer signed a global agreement with OPKO to develop and commercialize somatrogon for the treatment of GHD.
agreement, OPKO is responsible for implementing clinical projects and Pfizer is responsible for product registration and commercialization.
will assess the likelihood of other childhood and adult adaptations, as appropriate.
"The FDA's acceptance of our application is an exciting step, and our goal is to provide GHD children with a long-lasting, weekly treatment option," said Dr. Brenda Cooperstone, Chief Development Officer for Rare Diseases, Pfizer's Global Product Development Division.
if approved, somatrogon will help reduce the burden of daily growth hormone injections for children, their loved ones and caregivers.
Pfizer has been working for 35 years to improve the prognostication of GHD patients, somatrogon is another example of how we strive to positively impact patient quality of life and treatment compliance to help ensure that these patients reach their full potential.
" application is supported based on the results of a Phase 3 clinical trial.
was a randomized, open-label, positive drug-controlled trial conducted in more than 20 countries, in which 224 children with GHD who had not previously been treated were treated.
study, these patients were randomly assigned to two treatment groups in a 1:1 ratio: somatrogon (0.66 mg/kg, given once a week) and Genotropin (0.034 mg/kg, given once a day).
end of the trial was the treatment of 12 months of height growth rate.
end points include changes in height standard deviation over 6 and 12 months, safety, and pharmacological indicators.
who completed the study had the opportunity to participate in a global, open-label, multi-center, long-term expansion study in which patients could continue to receive or switch to somatrogon treatment.
95 percent of patients have been transferred to open label extension studies and received somatrogon treatment.
results showed that the study reached the main endpoint of non-poor effectiveness: 12 months of treatment, the minimum mean of the somatrogon group (10.12 cm/year) was higher than that of the Genotropin group (9.78 cm/year). the therapeutic difference in height growth rate (cm/year) was 0.33 (two-sided 95% confidence interval: -0.39, 1.05).
the somatrogon group had a higher change in height standard deviation score (key secondary endpoint) at 6 and 12 months than the Genotropin group.
addition, at 6 months, the somatrogon group had a higher change in height growth rate than the Genotropin group, another key secondary endpoint.
in clinical settings, these commonly used growth measurement methods are used to measure the potential for height growth of peers who may experience age-chasing and gender-matching.
the study, somatrogon's tolerance was generally good, with the type, number and severity of adverse events observed between treatment groups comparable to the once-a-day growth hormone Genotropin.
original source: US FDA ACCEPTS REGULATORY SOURCE FROM PFIZER and OPKO FOR REVIEW OF SOMATROGON TO TREAT PATIENTS WITH GROWTH RESULTS