The PBAC has rejected a twice-daily medication for Cushing’s syndrome for the second time because of its cost, despite acknowledging it was an effective therapy and there was a high clinical need.

Osilodrostat (Isturisa) was approved by the TGA in 2022 to treat adults with endogenous Cushing’s syndrome, but the oral non-steroidal cortisol synthesis inhibitor is yet to be sold in Australia.

The first attempt at a PBS listing was knocked back in early 2023 due to the “highly uncertain” cost-effectiveness and long-term benefits claimed by sponsor Recordati Rare Diseases Australia.

Its submission had requested for osilodrostat 1mg, 5mg and 10mg to be subsidised for patients who were not eligible to undergo pituitary gland surgery or for whom surgery was not curative.