Sangamo Therapeutics(US:SGMO) Globenewswireยท2025-09-04 12:05Core Insights - The data supports the potential of isaralgagene civaparvovec as a one-time, durable treatment for Fabry disease, offering significant multi-organ clinical benefits compared to current care standards [1][6] - The STAAR study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all patients, which the FDA has agreed will be the primary basis for approval [1][6] - Sangamo plans to submit a Biologics License Application (BLA) in 2026 under the Accelerated Approval pathway [1][6] Efficacy - The STAAR study included 32 dosed patients with a positive mean annualized eGFR slope of 1.965 mL/min/1.73m/year at 52 weeks, which is favorable compared to approved Fabry treatments [4] - At Week 104, a mean annualized eGFR slope of 1.747 mL/min/1.73m/year was observed for 19 patients [4] - Stable cardiac function was maintained over at least one year, with various cardiac metrics remaining stable [4] Safety - Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile, with most adverse events being grade 1-2 [12] - The most common treatment-emergent adverse events included pyrexia (60.6%), COVID-19 (36.4%), headache (30.3%), and nasopharyngitis (33.3%) [12] - All treatment-emergent adverse events resolved with clinical management, and there were no safety-related study discontinuations or deaths [12] Regulatory Designations - Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [8] - The company is preparing for BLA submission while engaging in business development for potential commercialization [8] Study Design - The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec in Fabry disease patients [10] - The study enrolled male and female patients, with a median age of 42 and a median follow-up duration of 24 months [7] Quality of Life Improvements - Statistically significant improvements in quality of life scores were observed, including role-physical (+14.8), vitality (+9.6), and bodily pain (+9.0) at week 52 compared to baseline [12] - Improvements in disease severity were reported in the Mainz Severity Score Index, with 22 patients showing total score improvements at 12 months [12]