Pfizer Inc. and OPKO Health Inc. announced on Tuesday that the European Commission has granted marketing authorization for the next-generation long-acting recombinant human growth hormone NGENLA (somatrogon), a once-weekly injection to treat children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. NGENLA provides pediatric patients, their caregivers and healthcare providers with a new treatment option for growth hormone deficiency (GHD) that reduces the frequency of required injections from once-daily to once-weekly.
“Growth hormone deficiency takes a substantial toll on children living with this rare disease and their families, and for many years, daily injections have been the standard-of-care, adding to the challenges they face. With NGENLA, we are proud to continue to advance the care of children in Europe who are impacted by growth hormone deficiency with a new, longer-acting option that significantly reduces treatment burden from once-daily to once-weekly injections,”said Reda Guiha, Regional President for International Developed Markets, Pfizer Rare Disease.
GHD is a rare condition defined by insufficient pituitary gland secretion of the growth hormone somatropin, which affects one in every 4,000 to 10,000 children globally. Genetic mutations can cause the disease, or it can be acquired after birth. Affected children will have continuous growth attenuation and an extremely small adult height if they do not receive treatment, and puberty may be delayed. Other issues with physical health and mental well-being may arise in children.
“By helping to minimize disease management burden, we believe NGENLA has the potential to improve quality of life for children impacted by growth hormone deficiency and their families, as well as increase treatment adherence, which can improve outcomes. We are pleased that the marketing authorization in the European Union will enable more children with growth hormone deficiency to benefit from once-weekly treatment,”said Phillip Frost, M.D., Chairman, and Chief Executive Officer, OPKO Health.
The results of a global, randomised, open-label, active-controlled Phase 3 study comparing once-weekly NGENLA to once-daily GENOTROPIN® were used to support the European Union’s (EU) marketing authorization of NGENLA (somatropin). The study’s primary endpoint of NGENLA non-inferiority to GENOTROPIN was satisfied after 12 months, as determined by yearly height velocity. In the research, NGENLA was usually well-tolerated and had a safety profile similar to GENOTROPIN.
NGENLA’s marketing authorisation is valid throughout the EU, as well as in Iceland, Norway, and Liechtenstein.