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Lyso-Gb3 is the circulating derivative of Gb3, the lipid that builds up when alpha-Gal A is deficient, and the main blood biomarker used to track Fabry disease activity. In FDA's written INTERACT feedback, plasma lyso-Gb3 was recommended as the primary pharmacodynamic readout for GT-GLA-S03. In the FACTS trial, plasma lyso-Gb3 fell 48% vs. no-ERT baseline (p<0.0001) at 5-year follow-up, with 4 of 5 patients below threshold at year 5.
Interpretation: Lyso-Gb3 is the most direct early signal that restored enzyme is doing its job biochemically.
Uncertainty: A biomarker is not an organ outcome. Lower lyso-Gb3 does not by itself show long-term protection of the kidneys, heart, or nervous system, which is why FDA also recommended longer eGFR follow-up (see Wednesday's post).
Read the underlying publication (PMID 36816757) for the full dataset.
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