The Food and Drug Administration (FDA) recently accepted Pfizer and OPKO Biologics Biologics License Application (BLA) for somatrogon for the treatment of pediatric growth hormone deficiency (GHD). Somatrogon belongs to a new class of long-acting growth hormones (GH) that are projected to steal substantial market share from the current leading GHD therapeutics, says GlobalData, a leading data and analytics company.
GHD is a rare endocrine disorder with a diagnosed prevalence rate of 0.014% in 2020 across the seven major markets (*7MM), according to GlobalData.
Fiona Chisholm, Pharma Analyst at GlobalData, comments: "This is an exciting time within the GHD space. For decades, the standard of care has been daily injections of recombinant human GH, which is associated with poor treatment compliance and suboptimal therapeutic outcomes. However, investment in the development of more convenient and user-friendly therapeutics has increased in recent years, with long-acting GHs at the forefront of this."
In August 2020, Novo Nordisk's Sogroya (somapacitan-beco) became the first long-acting GH to gain marketing authorization in GHD following FDA approval for the treatment of adult patients. Additionally, of the nine pipeline products in late-stage development (Phase II development and above) across the globe, six are long-acting GHs, according to GlobalData's Pharma Intelligence Center's Pipeline Products database. Besides somatrogon, another prominent pipeline candidate includes Ascendis Pharma's TransCon hGH (lonapegsomatropin). The FDA has accepted a BLA for TransCon hGH for the treatment of pediatric patients, and a marketing authorization application (MAA) for the treatment of pediatric GHD has been submitted to the European Medicines Agency (EMA).
Chisholm continues: "Long-acting GHs are administered on a weekly or biweekly, rather than a daily, basis, which dramatically reduces the injection burden for patients. This provides patients with a more manageable treatment strategy and could lead to improved treatment adherence."
Key opinion leaders (KOLs) interviewed by GlobalData consider poor treatment compliance to be the greatest unmet need in the market. Based on KOL insight, GlobalData anticipates strong uptake of long-acting growth hormones, with these drugs largely replacing the daily GHs.
Another research and development (R&D) strategy evident in the late-stage pipeline is the investment in alternative drug delivery routes. Lumos Pharma's Oratrope (ibutamoren mesylate) and Aeterna Zentaris' (macimorelin acetate) are orally administered ghrelin receptor agonists, both of which are in Phase II development. These drugs promote production of GH by mimicking the effects of a hormone that regulates GH secretion.
Chisholm adds: "This strategy again addresses the unmet needs for ease of use and convenience, which contribute to low treatment compliance in GHD. Currently, all marketed products are administered by injection, so an oral delivery route represents a strong commercial attribute."
*7MM: US, France, Germany, Spain, Italy, UK, and Japan