Listing Yorvipath: why a third PBAC submission must succeed

Palopegteriparatide (Yorvipath) is the first medicine that physiologically replaces the missing hormone in chronic hypoparathyroidism, and Australia's TGA was the first regulator worldwide to approve it after the FDA — yet two PBAC rejections in 2025 have left roughly 6,400–9,800 Australian adults tethered to a seventy-year-old calcium-and-vitamin-D regimen that controls a blood test while driving the kidney damage, neuropsychiatric symptoms and quality-of-life impairment documented in every major international cohort. The PaTHway trial now has three-year data showing 96% of patients remain independent of conventional therapy and mean eGFR has risen by 8.8 mL/min/1.73 m², with the largest gains in those with the worst baseline kidney function; Germany, France, the UK, Japan and the US have all moved to reimbursement while Australia has not. The PBAC's concerns were legitimate — ICER, surrogate endpoints, time horizon and limited Australian data — but each is answerable through instruments the Committee has itself built for rare-disease decisions: a confidential Special Pricing Arrangement, a Risk-Sharing Arrangement with expenditure caps, narrow initiation criteria aligned with the 2025 ESE guideline, explicit stopping rules, and a sponsor-funded Australian Hypoparathyroidism Registry. The article makes a respectful, specific case for a third submission framed as a Managed Access Program modelled on the Zolgensma and ivacaftor precedents, and calls on clinicians, patient organisations and affected Australians to coordinate their comment at the next consultation window.