Precision Drug for Fabry Disease Gains Approval in Argentina

According to a story from, the drug company Amicus Therapeutics recently announced that Argentina’s Agency of Medicines, Food, and Medical Devices (ANMAT) has granted approval for the drug migalastat (marketed as Galafold) as a treatment for Fabry disease patients that are sixteen years or older who have a suitable mutation. This approval will make the drug the only orally available precision therapy for Fabry disease in the country.

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.

Making History

Amicus has made plans to work through a local partner in the country, called Pint Pharma, in order to fulfill the remaining launch requirements for the drug in the next few months. This is the first Latin American country that has approved Galafold. Argentina is known to have at least 400 patients with Fabry disease in the country.

The drug is capable of acting on over 350 distinct mutations affecting the GLA gene per the Argentinean label for Galafold. The medication works by stabilizing the dysfunction of the affected enzyme which allows it to enter the lysosome, a cellular organelle that plays a critical role in the processing of sphingolipids.

Argentinean Fabry disease patients will now have access to one of the most effective treatments for their disease that is currently available anywhere in the world.

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