NEW THERAPY FOR CHILDREN WITH GROWTH HORMONE DEFICIENCY APPROVED IN AUSTRALIA

Rhea-AI Summary

Specialised Therapeutics (ST) has announced that SKYTROFA® (lonapegsomatropin) has received approval from Australia's Therapeutic Goods Administration (TGA) for treating pediatric growth hormone deficiency (GHD). This once-weekly injectable therapy is approved for children and adolescents aged 3-18 years with insufficient endogenous growth hormone secretion. GHD affects approximately 2,000 Australian children, occurring at a rate of 2-3 per 10,000 people. SKYTROFA utilizes Ascendis Pharma's proprietary TransConTM technology platform, which enables sustained release of active, unmodified somatropin over one week. The approval follows successful Phase 3 clinical trials (heiGHt, fliGHt, and enliGHten) involving over 300 pediatric patients. The therapy is distributed by ST under an exclusive agreement with Ascendis Pharma A/S covering several Asia-Pacific countries. Currently, SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS).

Positive

• FDA and EMA-approved therapy now available in Australia, expanding treatment options for GHD patients
• Once-weekly dosing versus traditional daily injections, improving convenience for pediatric patients
• Successful Phase 3 clinical trials with over 300 patients demonstrate efficacy
• Exclusive distribution rights secured for multiple Asia-Pacific markets

Negative

• Not currently listed on the Pharmaceutical Benefits Scheme (PBS), potentially limiting accessibility
• Presence of common adverse events including viral infections, fever, and other side effects

Insights

Pharmaceutical Regulatory Expert

Ascendis Pharma secures Australian approval for SKYTROFA, expanding its global footprint for this once-weekly GHD treatment.

The Australian regulatory approval of SKYTROFA (lonapegsomatropin) represents a significant milestone for Ascendis Pharma's global commercialization strategy. This marks the third major market approval for this product, following the US FDA approval in August 2021 and EMA approval in January 2022.

The Australian registration is particularly noteworthy as it's being commercialized through a strategic partnership with Specialised Therapeutics (ST), which holds exclusive distribution rights across seven Asia-Pacific markets including Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. This partnership model demonstrates Ascendis's strategic approach to global commercialization without requiring direct infrastructure investment.

SKYTROFA's competitive advantage stems from its innovative TransCon technology platform, which enables once-weekly dosing versus the standard daily injections typically required for growth hormone therapy. This represents a substantial improvement in the treatment burden for pediatric patients with growth hormone deficiency, a condition affecting approximately 2,000 children in Australia.

The approval is based on robust clinical evidence from three pivotal Phase 3 trials (heiGHt, fliGHt, and enliGHten) encompassing over 300 pediatric GHD patients. However, it's important to note that SKYTROFA is not currently listed on the Pharmaceutical Benefits Scheme (PBS) in Australia, which may impact initial uptake and access until reimbursement is secured.

For Ascendis Pharma, this approval expands the addressable market for SKYTROFA and validates their TransCon platform technology. The company now has regulatory clearance in three major regions (US, Europe, Australia), strengthening their position in the pediatric endocrinology space and potentially accelerating revenue growth through their partnership with ST in the Asia-Pacific region.

SKYTROFA^® (lonapegsomatropin) is now registered by the Therapeutic Goods Administration (TGA) for the treatment of paediatric growth hormone deficiency.^[1]

SINGAPORE, May 29, 2025 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics (ST) is pleased to announce the Australian registration of SKYTROFA® (lonapegsomatropin) as a new once-weekly injectable therapy for paediatric growth hormone deficiency (GHD).^[2] The Therapeutic Goods Administration (TGA) has approved SKYTROFA as a treatment for "growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion".^[1,2]

GHD is a rare disease affecting around 2-3/10,000 Australians, with approximately 2,000 children thought to be living with the condition.^[3] 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.^[3] 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.^[3] 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.^[3]

ST Chief Executive Officer, Mr Carlo Montagner, said the TGA approval of SKYTROFA demonstrated the company's ongoing mission to support patients with rare diseases in Australia and the Asia-Pacific region.

"We are delighted to have secured TGA registration of SKYTROFA for eligible Australian children with growth hormone deficiency. 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.^[4]

"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," said Mr Montagner.

SKYTROFA is being registered in Australia by ST, under an exclusive distribution agreement with biopharmaceutical company Ascendis Pharma A/S that covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam. The Australian registration of SKYTROFA follows approvals issued to Ascendis Pharma by the United States Food and Drug Administration (US FDA)^[5] in August 2021 and the European Medicines Agency (EMA)^[6] in January 2022.

Developed using Ascendis Pharma's proprietary TransCon^TM platform, SKYTROFA is delivered as a subcutaneous injection, available in various strengths.^[2] 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.^[2]

Ascendis Pharma's innovative TransCon^TM technology platform is designed to create new therapies with the potential to optimise therapeutic effect, including improving treatment efficacy, safety and dosing frequency.^[7] TransCon^TM molecules have three components: an unmodified parent drug, an inert TransCon™ carrier that protects it, and a TransCon^TM linker that temporarily binds the two.^[7]

SKYTROFA is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin.^[6] This allows the medicine to slowly release unmodified growth hormone into the body over the course of one week, removing the need for daily injections.

SKYTROFA consists of three components: the parent drug somatropin, an inert methyloxypolyethlene glycol carrier (mPEG), and a proprietary TransCon^TM linker that transiently binds the other two elements.^[8,9] While bound, the carrier inactivates the somatropin and shields it from renal excretion and receptor-mediated clearance.^[10] Following injection, autocleavage of the linker occurs under physiologic conditions, and SKYTROFA releases fully active, unmodified somatropin in a predictable manner.^[10]

The TGA registration of SKYTROFA was based on the results of Ascendis Pharma's three pivotal Phase 3 clinical trials, heiGHt, fliGHt and enliGHten, which collectively treated more than 300 paediatric patients diagnosed with GHD, including from Australia.^[2,8,11,12]

SKYTROFA was generally well-tolerated across all three clinical trials. The most commonly reported adverse events include viral infections, fever, cough, nausea and vomiting, haemorrhage, diarrhoea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels.

PBS Information: SKYTROFA is not listed on the Pharmaceutical Benefits Scheme (PBS).

About Specialised Therapeutics

Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases. ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas. ST is a member of the World Orphan Drug Alliance (WODA).

Additional information can be found at www.stbiopharma.com

About Ascendis Pharma

Ascendis Pharma is a global biopharmaceutical company focused on applying its innovative TransCon^TM technology platform to make a meaningful difference for patients. Guided by core values of Patients, Science, and Passion, and following its algorithm for product innovation, Ascendis Pharma applies TransCon^TM to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit: ascendispharma.com to learn more.

About Paediatric Growth Hormone Deficiency

Paediatric growth hormone deficiency (GHD) is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Physiological levels of growth hormone are required for overall endocrine health and development of healthy bone, muscle, and adipose tissue. Children with GHD are characterised by short stature and may also experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of human growth hormone (hGH) to improve growth and overall endocrine health.

About SKYTROFA (lonapegsomatropin)

Once-weekly SKYTROFA is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous growth hormone.^[8]

SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve children with growth hormone deficiency and those switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.^[2] The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).^[2]

SKYTROFA was studied in over 300 children with GHD across the Phase 3 program, which consisted of the HeiGHt Trial^[8] (for treatment-naïve patients), the FliGHt Trial^[11] (for treatment-experienced patients), and the EnliGHten Trial^[12] (a long-term extension trial). Patients who completed the HeiGHt or FliGHt Trials were able to continue in EnliGHten, with some on lonapegsomatropin treatment for over four years. Ascendis Pharma is also conducting the ongoing open-label portion of the global Phase 3 ForesiGHt Trial of SKYTROFA in adults with GHD.

Ascendis^®, TransCon^TM and SKYTROFA^®  are trademarks owned by the Ascendis Pharma group (NASDAQ: ASND).

About TransCon^TM Technologies

TransCon refers to "transient conjugation". Ascendis Pharma's innovative technology platform to create new therapies designed to potentially optimise therapeutic effect, including improving efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert TransCon carrier that protects it, and a TransCon linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g. pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied to a broad variety of therapeutics, such as antibodies, antibody fragments, proteins, peptides, or small molecules, and can be designed for systemic or local administration.

References:

[1] Therapeutic Goods Administration. Australian Register of Therapeutic Goods (ARTG). Available at: https://www.tga.gov.au/resources/artg?keywords=skytrofa. [Accessed May 2025]. [2] SKYTROFA (lonapegsomatropin) Product Information, Australia. [3] Australian Pituitary Foundation. Factsheet: A Guide To Growth Hormone Deficiency, 2023. Available at: https://pituitary.asn.au/wp-content/uploads/2023/09/APF-Growth-Hormone-Deficiency-Fact-Sheet-2023_Digital-Final.pdf [Accessed May 2025]. [4] Brod M et al. Qual Life Res. 2017 Jul;26(7):1673-1686. [5] US Food and Drug Administration (FDA). Drug Approval Package: SKYTROFA. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761177Orig1s000TOC.cfm [Accessed May 2025]. [6] European Medicines Agency (EMA). Skytrofa (previously Lonapegsomatropin Ascendis Pharma), January 2022. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/skytrofa [Accessed May 2025]. [7] Ascendis Pharma A/S. TransCon: A powerful technology platform central to our innovative approach. Available at: https://ascendispharma.com/technology/ [Accessed May 2025]. [8] Thornton PS, et al. J Clin Endocrinol Metab. 2021 Oct;106(11):3184-3195. [9] SKYTROFA® [package insert]. Palo Alto, CA: Ascendis Pharma, Inc. [10] Sprogøe K, et al. Endocr Connect. 2017;6(8):R171-r181. [11] Maniatis AK, et al. Horm Res Paediatr. 2022 Mar;95(3):233-243. [12] Maniatis AK, et al. J Clin Endocrinol Metab. 2022 Jun;107(7):e2680-e2689.

 

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SOURCE Specialised Therapeutics

FAQ

What is SKYTROFA and what was it approved for in Australia?

SKYTROFA (lonapegsomatropin) is a once-weekly injectable therapy approved by the TGA for treating growth failure in children and adolescents aged 3-18 years with growth hormone deficiency.

How many children in Australia are affected by growth hormone deficiency?

Approximately 2,000 Australian children are affected by growth hormone deficiency, with an incidence rate of 2-3 per 10,000 people.

What are the main advantages of SKYTROFA over existing treatments?

SKYTROFA's main advantage is its once-weekly dosing schedule, compared to traditional daily injections, using TransConTM technology for sustained release of growth hormone over one week.

Is SKYTROFA covered by the Australian PBS?

No, SKYTROFA is currently not listed on the Pharmaceutical Benefits Scheme (PBS) in Australia.

What are the common side effects of SKYTROFA treatment?

Common side effects include viral infections, fever, cough, nausea, vomiting, hemorrhage, diarrhea, abdominal pain, arthralgia, arthritis, and increased blood phosphate levels.