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# Fabry Disease Drug Development Guidance
- URL: https://www.fdaweb.com/fabry-disease-drug-development-guidance/
- Published: 2019-08-07T12:00:00.000Z
- Updated: 2026-09-15T01:42:36.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144712

FDA has issued a draft guidance, *Fabry Disease: Developing Drugs for Treatment*, to give sponsors recommendations regarding clinical trial design features that can support approval of drugs and biological products intended to treat Fabry Disease (FD). The [document](https://www.fda.gov/regulatory-information/search-fda-guidance-documents/fabry-disease-developing-drugs-treatment-guidance-industry?ref=fdaweb.com) says FD is a rare, X-linked, slowly progressive, lysosomal storage disorder caused by pathogenic variants in the galactosidase alpha gene resulting in absent or deficient activity of the lysosomal enzyme a-galactosidase A.

Contents of the guidance are Introduction, Background, and Key Considerations for Clinical Trials (eligibility criteria and trial design and efficacy endpoints).