FDA Accepts Sangamo's Request For Rolling BLA Submission For Fabry Disease Gene Therapy Candidate

(RTTNews) - Sangamo Therapeutics, Inc. (SGMO) on Friday announced that the U.S. Food and Drug Administration (FDA) has accepted its request for a rolling submission and review of the Biologics License Application (BLA) for isaralgagene civaparvovec, the company's gene therapy candidate for the treatment of adults with Fabry disease.

Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha (GLA) gene, leading to progressive damage to multiple organs, including the kidneys, heart, nerves, eyes, gastrointestinal system and skin.

Sangamo said results from its Phase 1/2 STAAR study demonstrated the potential for isaralgagene civaparvovec to serve as a one-time, durable treatment targeting the underlying pathology of Fabry disease, offering multi-organ clinical benefits that exceed current standards of care. The study also showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all treated patients. The FDA has agreed that this endpoint will serve as the primary basis for approval.

Isaralgagene civaparvovec has received 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, and Innovative Licensing and Access Pathway from U.K. Medicines and Healthcare products Regulatory Agency.

Sangamo said it plans to begin the rolling submission of the BLA to the FDA under the accelerated approval pathway later in the fourth quarter of 2025.