Clinical DataThe totality of data are supportive of approval, including the significant reduction in galactitol and consistent improvements across several clinical outcomes, which are now strengthened with the updated cognition data analysis.
Mechanism And BiomarkersThe effect on a biomarker (galactitol) is considerable with an established relationship with disease severity.
Regulatory ProgressTentative AdCom scheduling in galactosemia is a positive step in advance of PDUFA.