Phase 3 BRIGHT Study in Patients With Fabry Disease

Jack Johnson, Co-Founder and Executive Director of the Fabry Disease Support and Information Group, and VP of the Fabry International Network, discusses the phase 3 BRIGHT study testing Elfabrio (pegunigalsidase alfa-iwx) in patients with Fabry disease.

Fabry disease is a rare lysosomal storage disease caused by a deficiency of the enzyme alpha-GAL which helps break down the fatty acid, GL3. Without enough alpha-GAL, the lysosomes become filled with GL-3 and can not work well. Symptoms of Fabry disease may include episodes of pain, especially in the hands and feet, angiokeratomas, hypohidrosis, corneal opacity, and hearing loss. Internal organs, such as the kidney, heart or brain, may also be affected, leading to progressive kidney damage, heart attacks, and strokes. Fabry disease is caused by genetic changes in the GLA gene.

The BRIGHT study is a phase 3, open-label, multinational, switchover, 12-month study evaluating the pharmacokinetics, safety and efficacy of 2 mg/kg pegunigalsidase administered every four weeks. Pegunigalsidase is a PEGylated α-Gal A enzyme replacement therapy that is approved at 1mg/kg every two weeks for the treatment of adults with Fabry disease.

29 patients completed the study. No new safety concerns were observed and pegunigalsidase alfa median plasma concentrations were above the lower limit of quantification after each 4-week dosing interval. Median estimated glomerular filtration rate remained stable with change from baseline of -1.9 mL/min/1.73m2/year after 12 months of treatment. Additionally, plasma globotriaosylsphingosine concentrations were low and stable in women involved, with men demonstrating a slight increase compared to baseline.