Australia Approves First Chronic Hypoparathyroidism Treatment

The approval of YORVIPATH® (palopegteriparatide) by the Therapeutic Goods Administration (TGA) marks a significant advancement in the treatment of chronic hypoparathyroidism for adults in Australia. This announcement was made by Specialised Therapeutics (ST), an independent biopharmaceutical organization, which is bringing this novel therapy to the Australian market. YORVIPATH, a first-in-class parathyroid hormone (PTH) replacement therapy, is now the only medication registered in Australia for addressing this condition.

Chronic hypoparathyroidism is a rare and complex endocrine disorder that affects the endocrine system, with an estimated global prevalence of 6.4 to 37 people per 100,000. The disease results from inadequate production or absence of parathyroid hormone, leading to low calcium levels and high phosphorous levels in the blood. These imbalances can cause various symptoms, including muscle spasms, weakness, headaches, and cognitive issues, alongside potentially severe short-term and long-term health complications such as neuromuscular irritability and renal problems.

Professor Peter Ebeling, a leading endocrinologist in Australia, emphasized the significance of the TGA's approval, highlighting the innovation YORVIPATH brings to a field where treatment options have been limited. Prior to YORVIPATH, treatment relied heavily on calcium and active vitamin D supplementation to manage symptoms, which did not address the root cause of the PTH deficiency and posed a significant pill burden on patients. YORVIPATH offers a direct replacement for PTH, administered subcutaneously once daily, maintaining physiological levels of the hormone throughout the day.

The approval in Australia follows comprehensive studies, notably the Phase 3 PaTHway trial conducted by Ascendis Pharma. This trial demonstrated the efficacy and safety of YORVIPATH, showing that a substantial portion of participants achieved independence from conventional therapies like vitamin D and calcium supplements. The study also reported that YORVIPATH was generally well-tolerated, with the most common side effects being injection site reactions and headaches.

Beyond the medical benefits, the introduction of YORVIPATH is expected to improve the quality of life for those living with chronic hypoparathyroidism in Australia. The condition significantly impacts patients' daily lives and work productivity, and the new treatment promises to alleviate some of these burdens by addressing the disorder more effectively.

The Australian Thyroid Foundation's CEO, Beverley Garside, acknowledged the approval as an essential step forward but stressed the importance of securing subsidized access to ensure the treatment is affordable and accessible to all eligible patients. The goal is to have YORVIPATH covered under the Pharmaceutical Benefits Scheme (PBS), which would facilitate its availability to more patients in need.

Specialised Therapeutics is distributing YORVIPATH in Australia through an exclusive agreement with Ascendis Pharma, which covers several regions including New Zealand and parts of Southeast Asia. This collaboration aims to make the therapy widely accessible, leveraging Ascendis Pharma's innovative TransCon technology, which underpins YORVIPATH's sustained-release formulation.

Roy Khoury, Vice President at Ascendis Pharma, expressed satisfaction with the partnership, noting that it aligns with their mission to address unmet medical needs for rare diseases. Carlo Montagner, CEO of Specialised Therapeutics, reiterated the company's commitment to working with the Australian government and the community to ensure that YORVIPATH is available through the PBS rapidly.

The upcoming review by the Pharmaceutical Benefits Advisory Committee (PBAC) will be a critical next step in YORVIPATH's journey to becoming a mainstream treatment option for chronic hypoparathyroidism in Australia. If successful, it will be a milestone achievement in providing comprehensive care for this complex condition, offering hope and improved standards of living for many patients.