Sangamo announces alignment with FDA on ST-920 accelerated approval pathway

Sangamo (SGMO) announced the outcome of a recent interaction with the FDA, providing a clear regulatory pathway to accelerated approval for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease. The FDA has agreed in a Type B interaction that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the accelerated approval program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. The complete dataset to support an accelerated approval pathway will be available in the first half of 2025. This approach unlocks a potential BLA submission in the second half of 2025, three years ahead of previous estimates, and avoids the requirement for an additional registrational study to establish clinical efficacy. Sangamo engaged with the FDA on alternative pathways to potential approval following analysis of clinical data from the Phase 1/2 STAAR study showing encouraging safety and efficacy data, including promising preliminary evidence of improved kidney function. Based on these latest data, the FDA agreed that eGFR slope at 52 weeks can serve as an intermediate clinical endpoint to support a potential accelerated approval. The FDA also advised that eGFR slope at 104 weeks may be assessed to verify clinical benefit.