The European Medicines Agency (EMA) has issued a negative opinion on a request to approve a less frequent and higher-dose regimen of Elfabrio (pegunigalsidase alfa), an approved enzyme replacement therapy (ERT) for adults with Fabry disease. The request, submitted by Elfabrio’s developers, Chiesi Global Rare Diseases and Protalix…
Inflammation, believed to be a secondary or late complication, may instead be an early and active driver of Fabry disease, according to data from a small study in Spain. Researchers found ongoing immune activation and signs of inflammation in all patients, even in those with little buildup of the…
Treatment with Fabrazyme (agalsidase beta) for Fabry disease was safe and tolerated well over about a year, led to reductions in disease-related biomarkers, and eased symptoms in most patients, according to results of a post-marketing surveillance trial in China. An approved enzyme replacement therapy (ERT) for Fabry disease,…
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