According to a story from The Sydney Morning Herald, the Australian government recently announced that it will be adding Galafold, a drug used to treat rare Fabry disease, to the nation’s Life Saving Drugs Program. Galafold can prevent Fabry disease patients from facing an early death. The move allows patients in the country to access the drug free of charge.
About Fabry Disease
Fabry disease is a rare genetic disorder that primarily affects the heart, skin, and kidneys. As a lysosomal storage disease, it is characterized by a deficiency in the enzyme responsible for processing sphingolipids, which accumulate in the body as a result. The disorder is caused by mutations of the GLA gene. Symptoms include pain (which can affect the extremities, the entire body, or the digestive tract), kidney dysfunction, abnormalities of the heart valve and heart rhythms, fatigue, inability to sweat, and angiokeratomas (small red dots that appear on the skin). Treatments include enzyme replacement therapy, treatments to address organ specific problems, and Galafold. Galafold is effective in roughly 50 percent of patients, and only works for patients with certain types of mutations. Enzyme replacement therapy can help partially halt or reverse disease progression. To learn more about Fabry disease, click here.
Improved Access Means Saved Lives
Without coverage under the Life Saving Drugs Program, it was rather difficult for Fabry disease patients to get access to Galafold; out of pocket, this therapy can cost hundreds of thousands of dollars per year. One patient in Australia that has witnessed the benefits of this treatment first hand is Lea Chant. She first took the drug as a participant in a clinical trial, and she has continued to use it for eight years since then. Her son Christopher was also diagnosed with Fabry disease when he was just six months old.
Lea got involved in the trial not only to save her own life, but also that of her son’s. Christopher inherited the same mutation that Lea has, so if the drug worked for her, it should work for him as well. There are two of around 300 people in Australia that have been diagnosed with Fabry.
Galafold is the 14th drug to be added to the Life Savings Drugs Program.