Source – Pfizer
On 29 June 2023, Pfizer and OPKO Health jointly announced that the US Food and Drug Administration (FDA) has granted approval for NGENLA (somatrogon-ghla), a revolutionary once-weekly analog of human growth hormone. This innovative treatment is indicated for pediatric patients aged three years and older who suffer from growth failure caused by insufficient secretion of endogenous growth hormone. NGENLA is expected to be available for prescription in the United States starting from August 2023.
Pediatric growth hormone deficiency (GHD) is a rare condition characterized by inadequate secretion of somatropin, the growth hormone, from the pituitary gland. It affects approximately one in every 4,000 to 10,000 children. Without proper treatment, children with GHD experience persistent growth impairment, significantly short stature in adulthood, and delayed puberty. Furthermore, these children may face challenges related to their physical health and mental well-being.
“For more than 30 years, Pfizer has been committed to supporting children and adults living with growth hormone deficiency, beginning with the delivery of a medicine that has long been a part of the standard of care. We are excited to bring this next-generation treatment to patients in the United States, continuing our commitment to helping children living with this rare growth disorder reach their full potential.”
– Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer
The FDA approval is based on findings from a Phase III study, conducted across multiple centers, which assessed the safety and efficacy of NGENLA compared to once-daily somatropin. The study successfully met its primary endpoint, demonstrating that NGENLA is non-inferior to somatropin in terms of annual height velocity at the 12-month mark. NGENLA exhibited a favorable tolerability profile and demonstrated a safety profile similar to somatropin.
“The approval of NGENLA will be significant for children with growth hormone deficiency in the US. It holds potential to reduce the treatment burden that can come with daily growth hormone injections. As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, NGENLA could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”
– Joel Steelman, M.D., Pediatric Endocrinologist, Cook Children’s Health Care System
“Throughout our collaboration with Pfizer, we have worked tirelessly toward our shared goal of helping children living with growth hormone disease and their families. We are proud of the clinical development program that supported the FDA approval of NGENLA and are excited about its potential for these patients and their families as it becomes available in the United States.”
– Phillip Frost, M.D., Chairman and Chief Executive Officer, OPKO Health
NGENLA has already received approval for the treatment of pediatric GHD in over 40 markets, including Canada, Australia, Japan, and various European Union Member States. This FDA approval marks a significant milestone in the field of pediatric endocrinology, offering healthcare professionals and patients a convenient once-weekly treatment option for pediatric growth hormone deficiency.
