NEW YORK – Sangamo Therapeutics is hoping to submit a biologics license application to the US Food and Drug Administration in 2026 and seek approval to market its gene therapy for Fabry disease, ST-920 (isaralgagene civaparvovec).
The Richmond, California-based firm on Tuesday said that a "productive" type B meeting with the FDA to discuss development and next steps for ST-920 has given it clarity around process validation, commercial specifications, and a commercial launch manufacturing site. Based on this meeting, Sangamo has confidence to project the first quarter of 2026 as the time frame for a planned BLA submission for ST-920, which could facilitate approval and commercialization in the second half of the year.
Fabry disease is caused by mutations in the GLA gene and is characterized by the buildup of globotriaosylceramide, a lipid that then damages the kidneys, heart, nerves, eyes, gut, and skin. Renal deficiencies, marked by faster declines in the estimated glomerular filtration rate (eGFR), occur in patients with Fabry disease and can lead to end-stage renal disease and early death.
The firm is evaluating ST-920 in the Phase I/II STAAR study, in which all 32 dosed patients have at least 52 weeks of follow-up, data the FDA had required for the gene therapy to be reviewed under the accelerated approval pathway. According to a preliminary analysis, "the mean eGFR slope continued to remain positive" at the most recent cutoff date. Sangamo is expecting a pivotal data readout from the trial by the end of the second quarter.
"Following last year's alignment with the FDA on an accelerated approval regulatory pathway for ST-920, we are excited to have now gathered the one-year mean eGFR slope data that will serve as the primary efficacy endpoint for our planned BLA submission," Sangamo Chief Development Officer Nathalie Dubois-Stringfellow said in a statement. "Coupled with our recent productive FDA type B meeting, we have a clear regulatory pathway to a potential approval decision for ST-920, and we continue to advance BLA preparation activities."
Sangamo is also discussing a regulatory path for ST-920 with the European Medicines Agency and is negotiating a potential commercial agreement for the product in Fabry disease.