First targeted therapy in a decade to be approved for rare rheumatologic disease

Pharma News: The Therapeutic Goods Administration (TGA) has approved the first targeted therapy in a decade, granting orphan drug designation, for the treatment of a rare rheumatologic disease of grave significance.

CSL Vifor’s Tavneos (avacopan) in combination with a rituximab or cyclophosphamide-based regimen for the treatment of adults with anti-neutrophil cytoplasmic autoantibody (ANCA) – associated vasculitis (granulomatosis polyangiitis [GPA] or microscopic polyangiitis [MPA]); two main forms of AAV.

“This is a pleasing result, and we are committed to bringing this new treatment option to Australian patients living with AAV,” said James McDonnell, General Manager, CSL Vifor Australia and New Zealand.

“We expect to make a submission to the Pharmaceutical Benefits Advisory Committee as soon as possible, reinforcing our on-going commitment to improving the care of patients living with this rare disease.”

“This long-awaited new class of medication represents an important advancement for many patients living with AAV”, said Dr Daman Langguth, Director of Immunology at Sullivan Nicolaides Pathology and Clinical Immunologist at Wesley Hospital.

“We are confident that this novel therapy will provide more options to help control the disease, extend remission and meet major unmet medical needs in the treatment of AAV.”

The approval was based on the results of the phase-III ADVOCATE trial which was conducted in a total of 331 patients in 20 countries, including Australia. The study met its primary endpoints of disease remission at week 26 and sustained remission at week 52, as assessed by the Birmingham Vasculitis Activity Score (BVAS). The Tavneos-based regimen demonstrated superiority over standard of care at week 52. 

In Australia, Tavneos is commercialised under license from ChemoCentryx, a wholly owned subsidiary of Amgen, by Vifor Fresenius Medical Care Renal Pharma (VFMCRP) through CSL Vifor, with availability in the second half of 2023.

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