PBS Expands Listings for Fabry Disease, Kidney Cancer

Department of Health

Australians with Fabry disease, a rare genetic condition, and people with kidney cancer now have access to cheaper medicines under the Pharmaceutical Benefits Scheme (PBS).

Migalastat (Galafold), which has been available through the Life Saving Drug Program, will move to the PBS with wider eligibility criteria. It will be listed for the treatment of Fabry disease in patients 12 years and older who have certain genetic mutations and where the disease has affected their organs.

Fabry disease is caused by a missing or deficient enzyme which breaks down a particular type of fat. This leads to an abnormal build-up of fat causing pain in the hands and feet, dark red skin spots, hearing loss, digestive problems, alterations in the eye and life-threatening complications such as progressive kidney damage and heart disease.

This listing is expected to benefit around 65 patients each year. Without PBS subsidy, patients and their families might pay around $330,000 per year if treatment was accessed privately.

Cabozantinib (Cabometyx) will be expanded to include the treatment of nonclear cell renal cell carcinoma (RCC), a less common subtype of kidney cancer.

Cabozantinib is a targeted therapy which blocks the action of specific proteins, slowing the growth and cutting off the blood supply to the cancer.

Around 60 patients are expected to benefit from this expanded listing each year. Without the PBS subsidy, they might pay around $120,000 per course of treatment.

The PBS listings of these medicines means eligible patients will pay a maximum of $31.60 per script, or just $7.70 with a concession card.

Since July 2022, the Australian Government has approved extra funding for 235 new and amended listings on the PBS.

Quotes attributable to Minister Butler:

"The Albanese Government continues to make sure Australians have affordable access to the latest treatments.

"The listing of these medicines on the PBS gives patients and their doctors new options without the burden of worrying about the cost of treatment.

"It's part of our Government's commitment to keep medicines cheaper for Australians so they can live healthier lives, for longer."

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