New Treatment SKYTROFA for Pediatric Growth Hormone Deficiency Launched in Australia

New Therapy for Pediatric Growth Hormone Deficiency Approved in Australia

Introduction

In a groundbreaking advancement, Specialised Therapeutics (ST), an independent biopharmaceutical company, has secured registration for SKYTROFA® (lonapegsomatropin) by the Therapeutic Goods Administration (TGA) in Australia. This new medicine is aimed at children suffering from growth hormone deficiency (GHD), offering a game-changing treatment option for pediatric patients aged 3 to 18 years. With GHD currently affecting about 2 to 3 out of every 10,000 children in Australia, the approval of SKYTROFA represents hope for many families grappling with this rare condition.

Understanding Growth Hormone Deficiency

Growth hormone deficiency is a disorder where the pituitary gland fails to produce sufficient growth hormone, crucial for normal physical development during childhood. Characterized by stunted growth, it may also lead to delayed puberty and various health issues that markedly affect a child’s quality of life. Treatment strategies focus on restoring normal hormone levels to mitigate these symptoms, ultimately enhancing growth and overall well-being.

About SKYTROFA

SKYTROFA is a revolutionary therapy designed for once-weekly subcutaneous administration. This innovative prodrug of somatropin allows for sustained release of unmodified growth hormone, thus eliminating the need for daily injections. The medication is carefully formulated with the proprietary TransCon™ technology by Ascendis Pharma, enabling it to maintain therapeutic efficacy while improving the treatment experience. The approval of SKYTROFA in Australia complements its earlier endorsements from the FDA in the U.S. and the EMA in Europe.

How SKYTROFA Works

Developed using Ascendis Pharma's state-of-the-art TransCon™ platform, SKYTROFA incorporates a parent drug, an inactive carrier, and a linker that temporarily binds these two components. This mechanism shields the somatropin from premature clearance by the body until it's safely delivered and activated, ensuring consistent and controlled hormone release over a week.

Clinical Trials and Efficacy

The TGA registration was significantly supported by three pivotal Phase 3 clinical trials — heiGHt, fliGHt, and enliGHten — which collectively involved over 300 pediatric patients diagnosed with GHD. Results indicated that SKYTROFA was generally well-tolerated, demonstrating its potential effectiveness as a long-term treatment option.

Implications for Patients and Families

Mr. Carlo Montagner, CEO of ST, expressed his enthusiasm regarding the TGA approval, stating, "This pivotal moment underscores our commitment to supporting patients with rare diseases in Australia and the Asia-Pacific. Children with GHD face more than just growth challenges; they often encounter severe psychosocial consequences that can diminish their quality of life." This underscores the importance of condition-tailored therapies that not only address physiological growth but also promote general health and emotional well-being among young patients.

Future Prospects and Availability

SKYTROFA's entry into the Australian market marks another significant milestone for Specialised Therapeutics, following their recent launch of YORVIPATH® for another endocrine condition. The SKYTROFA injection will be available through an exclusive distribution agreement with Ascendis Pharma, covering multiple regions including Australia, New Zealand, and Southeast Asia. It will adhere to patient-specific dosing, with initial doses calculated according to body weight and adjusted based on treatment responses.

Conclusion

The approval of SKYTROFA in Australia is a promising development in the treatment of pediatric growth hormone deficiency, offering significant benefits for children and their families. This innovative therapy not only represents a scientific achievement but also brings hope to many who live with the challenging implications of GHD, paving the way for improved health outcomes and quality of life.

For more information on SKYTROFA or Specialised Therapeutics, please visit www.stbiopharma.com.