Sangamo's gene therapy for Fabry disease shows positive results in STAAR study.

Sangamo Therapeutics announced positive data from the STAAR study on isaralgagene civaparvovec, a gene therapy for Fabry disease. The therapy showed a favorable safety and tolerability profile and demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks. The FDA has agreed that the eGFR slope will serve as the primary basis for approval. Sangamo plans to submit a Biologics License Application in 2026 under the Accelerated Approval pathway.

Sangamo Therapeutics (Nasdaq: SGMO) has presented detailed data from its registrational Phase 1/2 STAAR study of isaralgagene civaparvovec (ST-920), a gene therapy for Fabry disease. The study, which enrolled 32 patients, demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope of 1.965 mL/min/1.73m2/year at 52 weeks across all dosed patients. This positive eGFR slope, along with stable cardiac function and sustained α-Gal A activity for up to 4.5 years, highlights the potential of ST-920 as a one-time, durable treatment for Fabry disease [1].

Key highlights of the study include the successful withdrawal of all 18 patients from Enzyme Replacement Therapy (ERT), indicating the therapy's ability to provide meaningful clinical benefits above current standards of care. The study also showed a favorable safety profile with mostly grade 1-2 adverse events, and no preconditioning was required [1].

Sangamo plans to submit a Biologics License Application (BLA) in Q1 2026 under the Accelerated Approval pathway, leveraging the positive data from the STAAR study. The FDA has agreed that the eGFR slope will serve as the primary basis for approval, underscoring the potential of ST-920 to address the underlying pathology of Fabry disease and provide transformative treatment benefits [1].

The STAAR study enrolled male and female patients who were either on ERT, ERT pseudo-naïve, or ERT-naïve, with a median age of 42 and a median duration of follow-up of 24 months. The longest treated patient achieved 4.5 years of follow-up, demonstrating the durability of the therapy [1].

Isaralgagene civaparvovec has received multiple regulatory designations, including Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency. Sangamo is also advancing BLA preparation activities and engaging in business development negotiations for a potential Fabry commercialization agreement [1].

Sangamo's pipeline includes multiple partnered programs and opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with them on LinkedIn and Twitter/X [1].

References:
[1] https://www.stocktitan.net/news/SGMO/sangamo-therapeutics-presents-detailed-data-from-registrational-fryvitr9hgsr.html