The FDA Just Approved the New Drug Application for Migalastat, a Treatment For Fabry Disease

According to an article from, the pharmaceutical company Amicus Therapeutics announced that the Food and Drug Administration (FDA) has the company’s New Drug Application for migalastat with filing for priority review. Migalastat is a meant for patients sixteen years or older who have Fabry disease.

Fabry disease is a rare, genetic, lysosomal storage disease. A mutation causes for a certain enzyme to be deficient, which leads to the build up of the glycolipid Gb3. This build up affects the functioning of various tissues and organs. Symptoms include pain in the extremities or digestive tract, kidney problems and failure, heart problems that cause an increased risk of heart disease and high blood pressure, papules on the skin, and an inability to sweat. While there are some treatments available, they can often be very costly. Fabry disease causes a reduced life expectancy, and many patients die from cardiovascular complications. In addition, many people eventually have to receive a kidney transplant. To learn more about Fabry disease click here.

Migalastat has already received both Fast Track and Orphan Drug designation from the FDA. Both of these designations will allow Migalastat to be developed more efficiently. Orphan Drug designation was specifically developed as an incentive for the development of rare disease treatments. It gives the company exclusive rights to development as well as a seven year market exclusivity period if the drug is approved and hits the market. The designation also waives certain fees to cut the cost of development. Orphan Drug designation is reserved for treatments that would fulfill an unmet medical need.

The treatment has also received approval for use in the European Union and in several other countries across the world. It is important to note that migalastat is only suitable for Fabry disease patients with ‘amenable’ mutations. While there are over 1,000 known GLA mutations associated with Fabry, the EU label recognized 348 that are classed as amenable, and therefore responsive to the drug. Data analysis suggests that the treatment should be effective for 35 percent to 50 percent of the current Fabry disease population. Migalastat is not intended for use with other forms of treatment such as enzyme replacement.

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