Sangamo Advances Gene Therapy for Fabry Disease With FDA's Acceptance of Rolling BLA Submission

2025-12-23 14:26:10

Abstract generation in progress

Sangamo Therapeutics, Inc. (SGMO) announced Friday that the U.S. Food and Drug Administration (FDA) has greenlit a rolling submission process for its Biologics License Application (BLA) evaluating isaralgagene civaparvovec, a groundbreaking gene therapy designed to address Fabry disease in adult patients.

Understanding the Disease Challenge

Fabry disease represents a serious lysosomal storage disorder stemming from mutations in the galactosidase alpha (GLA) gene. This genetic defect triggers progressive deterioration across multiple organ systems—kidneys, heart, nervous system, eyes, gastrointestinal tract, and skin—creating significant clinical burdens for patients. Current management options remain limited in their ability to halt disease progression comprehensively.

Clinical Evidence Supporting the Therapy

The Phase 1/2 STAAR study demonstrated compelling results for isaralgagene civaparvovec as a potential one-time intervention with durable effects. Notably, the trial showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at the 52-week mark across the entire treated patient cohort. This kidney function metric has been established by the FDA as the primary efficacy endpoint for regulatory approval. Beyond renal protection, the candidate displayed multi-organ clinical benefits that outperform existing treatment standards.

Regulatory Accelerations and Timeline

The therapeutic candidate has secured multiple expedited pathways: Orphan Drug designation, Fast Track status, and RMAT (Regenerative Medicine Advanced Therapy) designation from the FDA. International regulatory bodies have also provided endorsement through Orphan Medicinal Product classification and PRIME eligibility from the European Medicines Agency, alongside Innovative Licensing and Access Pathway recognition from the U.K. Medicines and Healthcare products Regulatory Agency.

Sangamo plans to commence the rolling BLA submission to the FDA via the accelerated approval route beginning in late 2025.

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