Less frequent Elfabrio dosing for Fabry disease appears safe, effective
BRIGHT study tested therapy in adults, given every four weeks at 2mg/kg
Treatment with Elfabrio (pegunigalsidase alfa), given at more spaced intervals and at a higher dose than that approved for Fabry disease, appears to be safe and effective for adults with stable disease, data from a Phase 3 clinical study suggests.
Developed jointly by Chiesi Global Rare Diseases and Protalix Biotherapeutics, Elfabrio is approved in the U.S., the European Union, and the U.K for infusion into a vein at a recommended dose of 1 mg/kg every other week. The goal of the BRIGHT (NCT03180840) study was to test if Elfabrio could be taken at less frequent intervals.
The study included a small number of patients, so more research is needed, but “Chiesi is committed to evaluating additional evidence to confirm the long-term results of this administration schedule,” Giacomo Chiesi, executive vice president of Chiesi Global Rare Diseases, said in a company press release.
The results of the study, “A phase III, open-label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies,” were published in the Journal of Inherited Metabolic Disease.
Fabry disease is caused by a deficiency in the alpha-galactosidase A (alpha-Gal A) enzyme, which results in fatty substances, such as globotriaosylceramide (Gb3), accumulating in various organs, leading to a range of symptoms and complications.
A different dosing schedule
A common treatment approach for Fabry disease is enzyme replacement therapy (ERT), where a version of the missing alpha-Gal A enzyme is supplied to help ease symptoms.
Elfabrio is a type of ERT for Fabry disease that’s been modified through a process called PEGylation, which helps the enzyme become more stable in the blood and stay in the body longer. The therapy is administered every other week at a dose of 1 mg/kg.
The BRIGHT study tested the safety and efficacy of Elfabrio when given every four weeks at 2mg/kg in adults with Fabry disease. The study included patients who’d been on other ERTs every two weeks for at least three years. Over the one-year study, the researchers focused on tracking any treatment-related side effects.
The study included 30 patients, the majority of whom were men, with most having previously used Fabrazyme (agalsidase beta). Elfabrio stayed at effective levels in the bloodstream throughout the four-week period. Nine patients had mild to moderate side effects, but none were severe. No new immune reactions developed, which was a positive outcome.
To check its effectiveness, the researchers monitored kidney health using a measure called eGFR. Kidney function stayed fairly stable throughout the study. They also tracked levels of a Fabry disease biomarker called lyso-Gb3, which stayed steady in the female participants, but slightly increased in some men with prior immune responses.
The findings suggest Elfabrio every four weeks is generally safe for adults with stable Fabry disease, but before less-frequent dosing can become a treatment option, more research is needed with a larger number of patients to confirm the results.
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