Specialised Therapeutics Secures Approval for YORVIPATH® in Chronic Hypoparathyroidism Treatment
Friday, February 21, 2025
Specialised Therapeutics (ST) has announced the approval of YORVIPATH® (palopegteriparatide) by the Therapeutic Goods Administration (TGA) for the treatment of chronic hypoparathyroidism in adults.
Hypoparathyroidism is a rare endocrine disorder affecting an estimated 6.4 to 37 per 100,000 people worldwide. It results from low or absent production of parathyroid hormone (PTH), leading to hypocalcaemia and hyperphosphataemia, which can affect nerve and muscle function.
Symptoms include muscle weakness, cramps, headaches, numbness, tingling, and cognitive issues. Severe complications can include renal problems, extraskeletal calcifications, and neuromuscular irritability.
YORVIPATH is a first-in-class PTH replacement therapy administered subcutaneously once daily. It is a prodrug of PTH [1-34] designed to deliver sustained PTH levels within the physiological range for 24 hours.
Approval in Australia is based on results from Ascendis Pharma's Phase 3 PaTHway trial, which involved 84 adults with chronic hypoparathyroidism. The most common side effects included injection site reactions, headaches, and paraesthesia.
The treatment has been granted Orphan Drug Designation and was assessed through the TGA’s Priority Review pathway. It is the first and only medicine registered on the Australian Register of Therapeutic Goods (ARTG) for this condition.
Until now, treatment options for chronic hypoparathyroidism have been limited to calcium and active vitamin D supplementation to maintain normal calcium levels. These therapies help manage symptoms but do not address the underlying PTH deficiency.
The introduction of YORVIPATH provides a new approach to treatment, offering an alternative to conventional therapies.
Beyond medical complications, hypoparathyroidism also impacts quality of life, affecting daily activities and work productivity. The Australian Thyroid Foundation (ATF) has acknowledged the significance of the TGA approval in improving health outcomes for individuals with chronic hypoparathyroidism.
However, access to subsidised treatment through the Pharmaceutical Benefits Scheme (PBS) remains a critical next step to ensure affordability.
YORVIPATH is distributed in Australia by Specialised Therapeutics under an exclusive agreement with Ascendis Pharma A/S, covering Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam.
The Australian approval follows recent regulatory approvals by the United States Food and Drug Administration (FDA) in August 2024 and the European Medicines Agency (EMA) in November 2023.
Source: stabiopharma.com
