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Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.
Fast Track designation aims to facilitate the development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Companies granted this designation are given the opportunity for more frequent interactions with the FDA. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met. The FDA has previously granted ST-920 Orphan Drug Designation.
“We are thrilled with the FDA’s decision to grant Fast Track Designation for ST-920. Fabry is a debilitating disease with life-long impact,” said Nathalie Dubois-Stringfellow, Ph.D, Sangamo’s Senior Vice President, Chief Development Officer. “This decision from the FDA underscores the potential for ST-920 to address a serious unmet need and serve as a meaningful therapeutic option for patients with Fabry disease. We are highly encouraged by this promising development and look forward to our expected meeting with the FDA on Phase 3 trial design in the summer.”
ST-920 is currently being evaluated in the Phase 1/2 STAAR study, with a total of 20 patients dosed to date. In February 2023, Sangamo announced promising results from the STAAR study via an oral presentation at the 19th Annual WORLDSymposium, showing sustained, elevated expression of alpha-galactosidase A (α-Gal A) activity in the 13 dosed patients as of the data cutoff, 78% globotriaosylceramide (Gb3) substrate clearance at 6-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, as measured by the SF-36 General Health survey. A copy of the presentation is available in the Presentations section of the Sangamo website. Sangamo is currently preparing for a potential Phase 3 trial and plans to meet with the FDA on the proposed Phase 3 study design in the summer, with a trial start anticipated by the end of 2023, depending on regulatory interactions.