Pfizer and OPKO’s Once-Weekly NGENLA™ (somatrogon) Receives EU Approval for Pediatric Growth Hormone Deficiency
Feb 16, 2022 01:52
CST Updated
01:52
Pfizer
Pharmaceutical R&D Developer
Opko
Pharmaceutical Product Developer
European Commission
The European Commission, abbreviated as the EU Commission, is a supranational body under the European Union. Within the EU political system, the European Commission primarily undertakes executive tasks, thus being roughly equivalent to the government in a national system. However, the European Commission has other functions as well. In particular, except for the few circumstances specified in the treaties, the European Commission is the only institution with legislative power in the EU legislative process.
Growth Hormone Deficiency in Children (Image Source: nationalchildrensresearchcentre.ie)
News on February 15, 2022 /BioValleyBIOON/ --PfizerPfizer and OPKO Health, Inc. recently announced jointly that the European Commission (EC) has approved the next-generation long-acting growth hormone Ngenla (somatrogon):This is a long-acting recombinant human growth hormone (hGH) injected once a week for the treatment of children with growth hormone deficiency (GHD).Ngenla is specifically indicated for: children and adolescents aged 3 years and older with growth disorders due to insufficient secretion of growth hormone, or GHD. In January this year, Ngenla also received approval in Japan. Ngenla will offer pediatric patients, caregivers, and healthcare providers a new treatment option for GHD, reducing the required injection frequency from once daily to once weekly.
Notably, in terms of U.S. regulation, in January 2022, the FDA issued a Complete Response Letter (CRL) regarding the Biologics License Application (BLA) for somatrogon. Pfizer is currently evaluating the FDA's feedback and will collaborate with...FDACollaborate to determine the appropriate path forward.
This EU approval is based on the results of a global, randomized, open-label, positive-controlled Phase 3 study. The study was conducted in children with GHD and compared the efficacy and safety of somatrogon (administered once weekly) with that of injectable recombinant human growth hormone Genotropin (somatropin, administered once daily). In this study,After 12 months of treatment, somatrogon was comparable to Genotropin in the primary endpoint of annual height velocity.In the study, somatrogon was generally well-tolerated, with a safety profile comparable to Genotropin. The types, numbers, and severity of adverse events observed were similar between the two treatment groups.
Growth Hormone Deficiency (GHD) is a rare condition characterized by insufficient secretion of growth hormone from the pituitary gland, affecting approximately one in every 4,000 to 10,000 children. In children, this condition may be caused by genetic mutations or acquired after birth. Due to inadequate levels of growth hormone (a hormone responsible for growth) secreted by the pituitary gland, children with GHD may experience stunted height and delayed puberty. Without treatment, affected children will continue to experience developmental delays into adulthood, resulting in extremely short stature. Additionally, children with GHD face other issues related to both physical and mental health.
For decades, the standard of care for GHD has been once-daily subcutaneous injections of human growth hormone (hGH) to improve growth and metabolic effects. The treatment burden of daily injections for caregivers and patients is high, which may lead to poor compliance and reduce overall treatment effectiveness.
Somatrogon is a new molecular entity containing the natural sequence of human growth hormone, with one copy of the C-terminal peptide (CTP) of the β-chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies at the C-terminus; the CTP can extend the half-life of this molecule.In the United States and the European Union, somatrogon has been granted Orphan Drug Designation (ODD) for the treatment of GHD in both children and adults. Research results to date indicate that, compared with daily hGH, weekly somatrogon significantly reduces lifestyle disruptions, supports patient preference, and improves adherence.
In 2014, Pfizer and OPKO entered into a global agreement to develop and commercialize somatrogon for the treatment of GHD. Under the agreement, OPKO is responsible for implementing the clinical program, while Pfizer handles product registration and commercialization. The two parties will evaluate the potential for other pediatric and adult indications as appropriate. (Bioon.com)