TGA gives green light to first medicine for hypoparathyroidism

The Therapeutic Goods Administration (TGA) has approved the first medicine indicated for treating chronic hypoparathyroidism in adults.

Specialised Therapeutics’ Yorvipath (palopegteriparatide) will be distributed under an exclusive agreement with Ascendis Pharma A/S across Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam.

Hypoparathyroidism, a rare endocrine disorder, results in abnormally low or absent production of parathyroid hormone (PTH), leading to low calcium levels (hypocalcaemia) and high phosphorous levels (hyperphosphataemia) in the bloodstream.

Professor Peter Ebeling AO, Endocrinologist and Head of the School of Clinical Sciences at Monash Health, said the approval of once daily Yorvipath subcutaneous injection addresses an unmet medical need.

“Until now, the only treatment option for adults with chronic hypoparathyroidism has been conventional therapy with calcium and active vitamin D supplementation to keep blood calcium levels normal,” said Professor Ebeling.

He further added, “While these treatments help to manage the symptoms of hypoparathyroidism, they do not address the underlying deficiency of PTH and contribute to the significant pill burden for patients. The lack of effective therapeutic options has been an urgent medical need in this area, and the local approval of Yorvipath represents an important advance in the treatment of chronic hypoparathyroidism in Australia.”

Results from the Phase 3 PaTHway trial demonstrated that 93% of patients treated with Yorvipath achieved independence from both active vitamin D and therapeutic doses of elemental calcium, significantly reducing their daily medication burden.

Beverley Garside, CEO of the Australian Thyroid Foundation, stated “While this is positive news, it is only the first step. For adults diagnosed with chronic hypoparathyroidism to truly benefit from this new treatment, it is essential they have the option of subsidised and affordable access to Yorvipath through the Pharmaceutical Benefits Scheme (PBS) as quickly as possible.”

The therapy will be reviewed by the Pharmaceutical Benefits Advisory Committee (PBAC) at its first meeting of the year in March.

Roy Khoury, Vice President and Head of International Markets at Ascendis Pharma, said “We are pleased to collaborate with Specialised Therapeutics in making this transformative therapy available, leveraging our TransCon technology for treating this debilitating rare disease.”

Specialised Therapeutics’ CEO, Carlo Montagner, said the approval of Yorvipath is a significant milestone following the recent FDA and EMA approvals.

“Chronic hypoparathyroidism has considerable and life-long wide-ranging impacts on these individuals and we are committed to working with the local hypoparathyroidism community and the Australian Government to ensure Yorvipath is accessible to eligible patients through the PBS at the earliest opportunity,” Montagner emphasised.

In reimagining healthcare across the entire patient journey, Health Industry Hub^TM is the only one-stop-hub uniting the diversity of the Pharma, MedTech, Diagnostics & Biotech sectors to inspire meaningful change.

The Health Industry Hub^TM content is copyright protected. Access is available under individual user licenses. Please click here to subscribe and visit T&Cs here.