YORVIPATH Becomes First Approved Treatment for Chronic Hypoparathyroidism in Australia

YORVIPATH: A Breakthrough in Chronic Hypoparathyroidism Treatment

On February 20, 2025, the Therapeutic Goods Administration (TGA) in Australia granted registration for YORVIPATH® (palopegteriparatide), marking it as a revolutionary treatment for adults suffering from chronic hypoparathyroidism. This condition is relatively rare, affecting between 6.4 and 37 individuals per 100,000 people globally and presents complex health challenges due to insufficient levels of parathyroid hormone (PTH).

Understanding Chronic Hypoparathyroidism

Chronic hypoparathyroidism is characterized by low PTH production from the parathyroid glands, leading to dangerously low calcium levels in the blood (hypocalcaemia) and excessive phosphorous levels (hyperphosphataemia). Symptoms can extend from muscle spasms and cramps to more severe issues such as cognitive impairment and renal complications. The conventional treatment has primarily involved calcium and vitamin D supplements, which, while helpful, fail to address the root hormonal deficiency and often result in a burden of daily medication for patients.

The Approval of YORVIPATH

YORVIPATH stands out as the first-in-class therapy that directly replaces PTH. It is administered subcutaneously once daily, aiming to maintain physiological PTH levels over a 24-hour period. The foundation of YORVIPATH's development lies in the findings from Ascendis Pharma's Phase 3 PaTHway trial. This well-structured, randomized, double-blind, placebo-controlled clinical trial, which included 84 adults over 26 weeks, showed significant efficacy and safety for YORVIPATH.

Notably, 93% of patients receiving YORVIPATH achieved independence from using both active vitamin D and calcium supplements, resulting in a reduced pill burden, a positive shift for many patients struggling with the current treatments. The tolerability profile was also encouraging, with the most common side effects being mild injection site reactions, headaches, and paraesthesia.

Voices from the Medical Community

Professor Peter Ebeling AO, a leading endocrinologist and head of the School of Clinical Sciences at Monash Health, emphasized the importance of this approval in addressing an urgent medical need. He stated that YORVIPATH represents a significant step forward in treating chronic hypoparathyroidism, providing an effective option that alleviates the limitations of previous treatment methods.

Beverley Garside, CEO of the Australian Thyroid Foundation (ATF), echoed this sentiment while highlighting the necessity for subsidized access through the Pharmaceutical Benefits Scheme (PBS). She noted the excitement around YORVIPATH's approval but cautioned that it was merely the first step toward ensuring affordable treatment for all affected individuals.

Next Steps for Patients and Accessibility

As YORVIPATH gains traction in Australia, it will undergo further scrutiny by the Pharmaceutical Benefits Advisory Committee (PBAC) during its earliest meeting from March 12-14, 2025. The listing on PBS following PBAC's approval is crucial for providing affordable access to patients.

Carlo Montagner, CEO of Specialised Therapeutics, recognized the registration of YORVIPATH as a pivotal moment for Australians dealing with hypoparathyroidism, while maintaining a commitment to continue working towards achieving a subsidized status that benefits eligible patients.

Conclusion

As the first approved medication for chronic hypoparathyroidism in Australia, YORVIPATH embodies hope for patients suffering from this challenging condition. With ongoing efforts aimed at ensuring its affordable availability through government programs, patients and healthcare providers are optimistic about the potential for YORVIPATH to transform the landscape of treatment for chronic hypoparathyroidism in Australia and beyond.