Assays for fabry disease treatment

A method of identifying a human subject suitable for a therapy for Fabry disease comprising measuring the presence of an anti-GLA neutralizing antibody in a biological sample of the subject, wherein the subject suitable for a therapy is an anti-GLA neutralizing antibody negative subject when a biological sample of the subject is analyzed, wherein the anti-GLA neutralizing antibody negative subject has a biological sample having lower than about 30% inhibition of α-Galactosidase A activity as measured by an anti-GLA neutralizing antibody assay.

A method of treating Fabry disease in a human subject in need thereof comprising administering a therapy for Fabry disease to the subject, wherein the subject is identified as an anti-α-Galactosidase A (GLA) neutralizing antibody negative subject when a biological sample of the subject is analyzed, wherein biological sample is a serum sample, which is diluted at minimum required dilution (MRD)15 or lower.

A method of identifying a human subject suitable for a therapy for Fabry disease comprising measuring the presence of an anti-GLA neutralizing antibody in a biological sample of the subject, wherein the subject suitable for a therapy is an anti-GLA neutralizing antibody negative subject when a biological sample of the subject is analyzed, wherein biological sample is a serum sample, which is diluted at MRD15 or lower.

A method of standardizing an anti-GLA neutralizing antibody assay comprising determining a GLA drug diluent concentration by measuring the effect of the GLA drug level on the inhibitory effect of an antibody designated as RP-01.

A method of treating Fabry disease in a human subject in need thereof comprising administering a non-enzyme replacement therapy for Fabry disease to the subject, wherein the subject is identified as an anti-α-Galactosidase A (GLA) neutralizing antibody positive subject when a biological sample of the subject is analyzed, wherein the anti-GLA neutralizing antibody positive subject has a biological sample having higher than about 10% inhibition of α-Galactosidase A activity as measured by an anti-GLA neutralizing antibody assay.

A method of identifying a human subject suitable for a non-enzyme replacement therapy for Fabry disease comprising measuring the presence of an anti-GLA neutralizing antibody in a biological sample of the subject, wherein the subject suitable for a therapy is an anti-GLA neutralizing antibody positive subject when a biological sample of the subject is analyzed, wherein the anti-GLA neutralizing antibody positive subject has a biological sample having higher than about 10% inhibition of α-Galactosidase A activity as measured by an anti-GLA neutralizing antibody assay.

A method of identifying a human subject who is not eligible for an enzyme replacement therapy for Fabry disease, comprising measuring the presence of an anti-GLA neutralizing antibody in a biological sample of the subject, wherein the sample having higher than about 10% inhibition of α-Galactosidase A activity as measured by the anti-GLA neutralizing antibody assay is identified as the anti-GLA neutralizing antibody positive sample, and wherein the subject not eligible for the enzyme replacement therapy for Fabry disease has the anti-GLA neutralizing antibody positive sample.