Yorvipath approved in Australia for chronic hypoparathyroidism
PTH replacement therapy will be available through Specialised Therapeutics
Yorvipath (palopegteriparatide) has been approved in Australia as a parathyroid hormone (PTH) replacement therapy for adults with chronic hypoparathyroidism.
The news follows similar approvals in the European Union in 2023, and in the U.S. last year. Yorvipath will be made available in Australia through Specialised Therapeutics, under an exclusive distribution agreement with Ascendis Pharma, which developed the therapy.
“The Australian approval of Yorvipath reflects our values and dedication to applying science to help address the significant unmet medical needs expressed by the hypoparathyroidism community,” Roy Khoury, head of international markets at Ascendis, said in a press release. “We are pleased to partner with Specialised Therapeutics to broaden access to this innovative therapy, based on our TransCon technology, which offers a new approach to the treatment of this often-debilitating rare disease.”
The treatment will soon be reviewed by the Pharmaceutical Benefits Advisory Committee, an independent expert body appointed by the Australian Government to recommend new medicines for listing on the Pharmaceutical Benefits Scheme.
“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 as quickly as possible,” said Beverley Garside, CEO of the Australian Thyroid Foundation.
Yorvipath helps manage blood calcium levels in adults with hypoparathyroidism
Yorvipath, previously known as TransCon PTH, is a long-acting PTH replacement therapy that contains palopegteriparatide, an inactive precursor molecule of PTH. It is used to manage blood calcium levels in adults with hypoparathyroidism, ease symptoms, and reduce the need for calcium and vitamin D supplements.
It is given once daily through a subcutaneous (under-the-skin) injection, with a sustained release that’s designed to provide PTH levels in the normal range over 24 hours.
Hypoparathyroidism is characterized by lower-than-normal levels of PTH, resulting in low calcium and high levels of phosphorus in the blood. This leads to a wide range of symptoms, including cramps, pain, twitching, and kidney stones.
Yorvipath’s approval was supported by data from the Phase 3 PaTHway trial (NCT04701203), which enrolled adults with hypoparathyroidism. Nearly 80% of Yorvipath-treated participants achieved normal blood calcium levels and independence from conventional therapy (meaning no vitamin D and no more than 600 mg of calcium supplementation), and did not require an increase in a Yorvipath dose in the last month of treatment. This contrasted with 5% of patients on a placebo achieving this composite goal. Treatment was generally well tolerated.
In the subsequent long-term extension study, the benefits have been generally sustained, including kidney function improvements at two years.
“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 Peter Ebeling, an endocrinologist and head of the School of Clinical Sciences at Monash Health in Australia. “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.”
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