PRX-102 (pegunigalsidase alfa) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with Fabry disease. Now branded Elfabrio (pegunigalsidase alfa-iwxj), the enzyme replacement therapy (ERT) is to be given as an intravenous, or into the vein, infusion every two weeks. It was codeveloped…
The first patient in a Phase 1/2 clinical trial of Fabry disease gene therapy candidate FLT190 has been dosed. Enrollment is ongoing at the Royal Free Hospital, in London, U.K. More information and contacts are available here. Fabry disease is caused by a faulty GLA gene, which provides…
One-time administration in mice of a gene therapy candidate known as ST-920 led to substantial increases in the activity of the key Fabry disease enzyme alpha-GalA and marked reductions in the levels of the fatty molecules that accumulate in patients with this disease. A Phase 1/2 clinical trial testing…
Treatment with the gene therapy candidate AVR-RD-01 showed sustained efficacy and was well-tolerated in Fabry disease patients regardless of prior enzyme replacement therapy (ERT), according to preliminary results of a Phase 1 and a Phase 1/2 trial. Avrobio’s AVR-RD-01 is a gene therapy that uses hematopoietic…
Treatment with Fabrazyme (agalsidase beta) leads to greater heart and biochemical improvements in Fabry disease patients than Replagal (agalsidase alfa), a study suggests. The study, “Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study,” appeared in the Journal of Medical Genetics. Fabry…
Two independent studies published in the journal Molecular Genetics and Metabolism and presented at the 14th Annual WORLDSymposium in San Diego, California, have discovered possible biomarkers to diagnose heart and kidney disorders in Fabry disease. One study, “Collagen type I synthesis biomarkers predict the progression of Fabry…
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