TGA approves therapy for paediatric growth hormone deficiency

Independent biopharmaceutical company Specialised Therapeutics (ST) has announced the Australian registration of its drug SKYTROFA (lonapegsomatropin) as a once-weekly injectable therapy for paediatric growth hormone deficiency (GHD). The Therapeutic Goods Administration (TGA) says it has approved SKYTROFA as a treatment for growth failure in children and adolescents aged from 3–18 years due to insufficient endogenous growth hormone secretion.

GHD is a rare disease affecting 2–3/10,000 Australians, with approximately 2000 children thought to be living with the condition. It occurs when the pituitary gland, located at the base of the brain, does not produce an adequate level of growth hormone, which is essential for promoting healthy growth in children.

In children, GHD is typically characterised by slow height growth, resulting in short stature, and may also include delayed puberty, impaired hair growth and headaches. The goal of treatment for GHD is to restore normal levels of growth hormone in the body, reduce physical symptoms, enhance metabolic health and improve quality of life.

SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin. This allows the medicine to slowly release unmodified growth hormone into the body over the course of the week, removing the need for daily injections. The starting dose depends on the patient’s body weight and is then adjusted individually by the treating endocrinologist based on the patient’s response to treatment.

SKYTROFA is being registered in Australia under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma — whose TransCon technology platform was used to develop the treatment — that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand and Vietnam. The TGA registration was based on the results of Ascendis Pharma’s three pivotal Phase 3 clinical trials, which collectively treated more than 300 paediatric patients diagnosed with GHD and found the drug was generally well tolerated.

“We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency,” said ST CEO Carlo Montagner. “Beyond short stature, children and adolescents with the condition may experience considerable physical and psychosocial impacts on their daily life, such as poor concentration, decreased strength or muscle development, fatigue, and reduced quality of life.

“This announcement also represents a significant milestone for Specialised Therapeutics, marking the second endocrinology therapy we have successfully registered in Australia, following the approval of YORVIPATH (palopegteriparatide) earlier this year.”

Image credit: iStock.com/Viktor Cvetkovic