Australians with Fabry disease, a rare genetic condition that can lead to alterations in the eye and life-threatening complications, 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.
The PBS listings of this medicine means eligible patients will pay a maximum of $31.60 per script, or just $7.70 with a concession card.
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