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Sangamo Therapeutics Initiates Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease
STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"STAAR study demonstrated positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across all dosed patients in the study, which U.S. Food and Drug Administration (FDA) has agreed will serve as an endpoint to support accelerated approval pathway Isaralgagene civaparvovec continues to show favorable safety and tolerability profile Sangamo expects to complete Biological License Application (BLA) submission under accelerated approval pathway in second quarter of 2026 RICHMOND, Calif., Dec. 18, 2025 (GLOBE NEWSWIRE) -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, has initiated a rolling submission of a BLA to the FDA seeking accelerated approval of isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease. Rolling submission allows for completed modules of the BLA to be submitted and reviewed by the FDA on an ongoing basis rather than waiting for the entire BLA to be submitted at once. “The initiation of our BLA submission marks an important milestone for Sangamo and for Fabry patients in need,” said Nathalie Dubois-Stringfellow, Ph.D., Sangamo’s Chief Development Officer. “The compelling data from our STAAR study shows the potential of ST-920 to provide safe and long-lasting clinical benefits to a wide range of Fabry disease patients. We look forward to working with the FDA as we continue to advance the regulatory process.” The totality of data from the registrational STAAR study demonstrated the potential for isaralgagene civaparvovec as a one-time, durable treatment of the underlying pathology of Fabry disease to provide meaningful, multi-organ, clinical benefits above current standards of care. Furthermore, the STAAR study demonstrated a positive mean annualized eGFR slope at 52-weeks across all dosed patients in the study, which the FDA has agreed will serve as endpoint to support accelerated approval. Isaralgagene civaparvovec has been granted Orphan Drug, Fast Track and RMAT designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency. Sangamo expects to complete submission of the BLA to the FDA under the accelerated approval pathway in th...