James Vane-Tempest's questions to SANGAMO THERAPEUTICS (SGMO) leadership • Q2 2025
Question
James Vane-Tempest of Jefferies & Company Inc. inquired about Sangamo's Fabry disease program, asking if a pre-BLA meeting with the FDA has occurred to discuss using one-year eGFR data for approval, and what additional data, such as meta-analysis details or individual patient trajectories, can be expected at the upcoming ICIEM conference.
Answer
CEO Sandy Macrae clarified that the pre-BLA meeting has not yet occurred but reiterated the FDA's prior agreement for an accelerated approval pathway based on one-year eGFR data. He stated the company has no expectation that the FDA will require anything beyond the one-year data. Chief Development Officer Nathalie Dubois-Stringfellow added that the upcoming ICIEM presentation will include more detailed data than the initial top-line press release, though individual patient data is unlikely to be shown at the conference.