Research & Development
Sangamo Therapeutics receives FDA Fast Track Designation for Fabry disease gene therapy
22 May 2023 -

Genomic medicine company Sangamo Therapeutics Inc (Nasdaq:SGMO) announced on Monday that the US Food and Drug Administration (FDA) has granted Fast Track Designation to isaralgagene civaparvovec (ST-920), a gene therapy candidate for Fabry disease.

This designation expedites the review process for therapeutics targeting serious or life-threatening conditions with unmet medical needs.

ST-920 is currently being evaluated in the Phase 1/2 STAAR study, with promising results demonstrating sustained, elevated expression of alpha-galactosidase A activity in the 13 dosed patients, 78% globotriaosylceramide substrate clearance at six months and 77% reduction in urine podocyte loss in one of the first kidney biopsies, and a clinically meaningful and statistically significant increase in mean general health scores.

Sangamo is preparing for a potential Phase 3 trial and plans to engage with the FDA on the study design in the summer, with a trial expected to start by the end of 2023.

Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene, resulting in deficient alpha-galactosidase A enzyme activity. The associated buildup of globotriaosylceramide in cells can lead to organ damage. Symptoms include skin blemishes, kidney disease, heart failure, vision problems and neuropathic pain.

Fast Track Designation provides Sangamo Therapeutics with increased FDA interactions and potential eligibility for Accelerated Approval and Priority Review. ST-920 has also received Orphan Drug Designation from the FDA and Orphan Medicinal Product designation from the European Medicines Agency.