According to a story from MD Magazine, the US Food and Drug Administration (FDA) has approved the medication migalastat, also known as Galafold, for the treatment of Fabry disease. This is the first oral medication for adult patients that has been approved in the US. Migalastat was first approved for treating Fabry disease by the European Union in 2016.
About Fabry Disease
Fabry disease is a type of rare, genetic lysosomal storage disease. It follows x-linked inheritance. The disease is caused by a mutation which leads to a deficiency of a certain enzyme that is responsible for breaking down a type of glycolipid called GL-3. As a result, this substance accumulates in various areas of the body, such as organs, body tissues, and blood vessels. Symptoms of Fabry disease include fatigue, pain, increased risk of stroke, heart problems, small skin lesions called angiokeratomas, and kidney dysfunction. Treatment involves enzyme replacement and more specialized care for organ complications. Cardiovascular disease is the most common cause of death; most patients will eventually have to receive kidney transplants. Life expectancy for patients is reduced, especially for males. To learn more about Fabry disease, click here.
The FDA previously rejected migalastat on the basis of insufficient data, but this new approval comes after a six month study that involved 45 patients with Fabry disease. In this study, patients treated with migalastat showed reduced GL-3 levels in comparison to placebo. The drug represents a new approach to treating this illness. While enzyme replacement is the most common treatment, migalastat instead increases the activity of the enzyme that is already present. Side effects of the drug are fairly mild and include nausea, fever, throat irritation, headache, and urinary tract infections.
The More The Merrier
With this new approval Fabry disease patients in the US will finally have access to a new treatment approach that has already been available in the EU for years. Like many rare diseases, treatment options for Fabry disease are very limited, so having more choices is always good news. In addition, migalastat offers an advantage to patients with quality of life concerns, since it is the first orally administered treatment available for Fabry disease.