RICHMOND, Calif. — 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 the second quarter of 2026.
About the STAAR Study
The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec, or ST-920, a gene therapy product candidate in patients with Fabry disease. Isaralgagene civaparvovec requires a one-time infusion without preconditioning.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), which leads to deficient alpha-galactosidase A (α-Gal A) enzyme activity, which is necessary for metabolizing globotriaosylceramide (Gb3). The buildup of Gb3 in the cells can cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. Symptoms of Fabry disease can include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain and tingling in the extremities.
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options. Sangamo believes that its zinc finger epigenetic regulators are ideally suited to potentially address devastating neurological disorders and that its capsid discovery platform can expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment. To learn more, visit www.sangamo.com and connect with us on LinkedIn and X.
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