Pfizer's long-acting growth hormone somatrogon filed for listing in Japan

OPKO Health recently announced that its partner Pfizer Japan has submitted a new drug application (NDA) to Japan's Ministry of Health, Labour and Industry (MHLW), a long-acting human growth hormone (hGH) that is used once a week to treat children with growth hormone deficiency (GHD).
month, Pfizer and OPKO announced that the FDA had accepted Somatrogon's Biological Products Licensing Application (BLA) and had designated a target date for the Prescription Drug User Charge Act (PDUFA) as October 2021.
if approved, somatrogon would provide a long-lasting, weekly treatment option that would help reduce the burden of daily growth hormone injections for GHD children, their loved ones and caregivers.
the Japanese application, based on the results of a Japanese Phase 3 study and a Global Phase 3 study.
the study, conducted in GHD children, compared the efficacy and safety of somatrogon (once a week) with the recombinant human growth hormone Genotropin (somatropin, given once a day).
two studies, after 12 months of treatment, somatrogon was comparable to Genotropin in terms of height growth rate in the main endpoint years.
two studies, somatrogon was generally well-to-do, as safe as Genotropin, and the types, numbers, and severity of adverse events observed between the two treatment groups were similar.
GHD is a serious rare disease caused by a deficiency of growth hormone secreted by 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.
, somatrogon's BLA is supported based on the results of Phase 3 clinical trials worldwide.
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 on a 1:1 scale: somatrogon (0.66 mg/kg, given once a week) and Genotropin (0.034 mg/kg, given once a day).
end point of the trial was the 12-month rate of height growth.
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 another key secondary endpoint than the Genotropin group, which had a higher change in height growth rate.
in clinical settings, these commonly used growth measurement methods are used to measure the potential for height growth of peers who may experience catch-up age 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.