The Food and Drug Administration (FDA) has approved Elfabrio® (pegunigalsidase alfa-iwxj) for the treatment of adults with confirmed Fabry disease.
Fabry disease is a rare genetic progressive disorder caused by deficient activity of the lysosomal α-galactosidase-A enzyme, which leads to progressive buildup of globotriaosylceramide (Gb3) in blood vessel walls throughout the body. Elfabrio is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. The product is internalized and transported into lysosomes where it is thought to exert enzymatic activity and reduce accumulated Gb3.
The approval was based on data from 2 clinical trials: an open-label dose ranging study (ClinicalTrials.gov Identifier: NCT01678898) in patients who were enzyme replacement therapy (ERT)-naïve or who had not received ERT for more than 26 weeks and had a negative test for anti-pegunigalsidase alfa-iwxj IgG antibodies; as well as a phase 3, double-blind, and active-controlled trial (ClinicalTrials.gov Identifier: (NCT03566017) in ERT-experienced adults diagnosed with Fabry disease.
In the phase 3 trial, patients were randomly assigned to receive at least 1 dose of pegunigalsidase alfa (n=52) or agalsidase beta (n=25). The primary efficacy endpoint was the annualized rate of change in estimated glomerular filtration rate (eGFR slope) assessed over 104 weeks.
Findings showed that pegunigalsidase alfa was noninferior to agalsidase beta in controlling eGFR decline. The estimated mean eGFR slope was -2.4 and -2.3mL/min/1.73 m2/year on pegunigalsidase alfa and agalsidase beta respectively. The estimated treatment difference was -0.1mL/min/1.73 m2/year (95% CI, -2.3, 2.1).
The most common adverse reactions (incidence greater than or equal to 15%) reported with pegunigalsidase alfa were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.
Elfabrio is supplied as a preservative-free solution in a single-dose vial. Each vial contains 20mg/10mL of pegunigalsidase alfa-iwxj. Treatment is administered by intravenous infusion every 2 weeks.
- Chiesi Global Rare Diseases and Protalix BioTherapeutics announce FDA approval of Elfabrio® (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease. News release. May 10, 2023, https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-and-protalix-biotherapeutics-announce-fda-approval-of-elfabrio-pegunigalsidase-alfa-iwxj-for-the-treatment-of-fabry-disease-301820680.html.
- Package insert. Chiesi; 2023. Accessed May 10, 2023. https://resources.chiesiusa.com/Elfabrio/ELFABRIO_PI.pdf.