Eric Wallace, MD, The University of Alabama at Birmingham, discusses an analysis of treatment comparisons between pegunigalsidase alfa versus other therapies for left ventricular mass index (LVMi) in Fabry disease.
Fabry disease is a rare lysosomal storage disease characterized by a deficiency of alpha-galactosidase (alpha-GAL). Alpha-GAL is important in the breaking down of globotriaosylceramide (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. Milder forms of Fabry disease may appear later in life and affect only the heart or kidneys. Fabry disease is caused by genetic changes in the GLA gene.
The objective of this study was to indirectly compare the mean change from baseline in LVMi for pegunialsidase alfa with agalsidase alfa and migalastat in patients with Fabry disease by network meta-analysis (NMA) and simulated treatment comparison (STC). Pegunialsidase alfa is a pegylated recombinant form of human alpha-GAL, indicated as an enzyme replacement therapy for treatment of patients with Fabry disease.
Observations from the analysis included unanchored STC results mostly consistent with that of the NMA. However, no statistical difference was observed between pegunigalsidase alfa versus agalsidase alfa, placebo, or migalastat. The NMA demonstrated a nominal trend favoring pegunialsidase alfa, but with no statistical significance. Previous studies suggest that pegunialsidase alfa is comparable to other Fabry treatment options in terms of change from baseline in LVMi.
To learn more about Fabry disease and other rare lysosomal storage disorders, visit https://checkrare.com/diseases/lysosomal-storage-disorders/
