The U.S. Food and Drug Administration (FDA) has approved pegunigalsidase alfa (Elfabrio), a plant-based enzyme replacement therapy with an initial half-life of 78.9 ± 10.3 hours, for the treatment of adult patients with Fabry disease.
Fabry disease is a rare X-linked lysosomal storage disorder that results in the cellular buildup of globotriaosylceramide. Characteristic features of Fabry disease include acroparesthesias, hypertrophic cardiomyopathy, proteinuria, gastrointestinal problems, angiokeratomas, hypohidrosis, corneal opacity, tinnitus, and hearing loss. Fabry disease also involves potentially life-threatening complications such as progressive kidney damage, heart attack, and stroke.
The approval was largely based on a clinical development program in which the safety, tolerability, and efficacy of pegunigalsidase alfa was evaluated in more than 140 patients with Fabry disease and followed for up to 7.5 years. The drug was studied in both ERT-naïve and ERT-experienced patients, including a trial that met its primary endpoint with pegunigalsidase alfa demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline, and in which pegunigalsidase alfa was generally well-tolerated.
To learn more about Fabry disease and other lysosomal storage disorders, visit https://checkrare.com/diseases/lysosomal-storage-disorders/