FDA announces draft guidance on developing drugs for Fabry disease

FDA announces draft guidance on developing drugs for Fabry disease

The U.S. Food and Drug Administration is announcing the availability of a draft guidance for industry titled, “Fabry Disease: Developing Drugs for Treatment." This draft guidance provides recommendations to sponsors regarding clinical trial design features that can support approval of drugs and biological products intended for the treatment of Fabry disease, a rare genetic disease that causes buildup of a certain type of fat within lysosomes.

There is an unmet need for further drug development for the treatment of Fabry disease. There are currently two drugs approved for Fabry disease, both are approved under accelerated approval. The FDA intends for the draft guidance to provide a path forward for drug development in Fabry disease.

The FDA seeks comments and suggestions on this guidance. For more information, please refer to the guidance.