Two-year data from Protalix BioTherapeutics’ Phase 1/2 extension trial of PRX-102 (pegunigalsidase alfa) show that the treatment prevented a decline in kidney and heart function in Fabry disease patients. People in this small, open-label study also reported a significant reduction in the severity of their symptoms. The data support…
The European Commission has granted Protalix BioTherapeutics’ experimental treatment PRX-102 (pegunigalsidase alfa) Orphan Drug Designation for the treatment of Fabry disease, the company recently announced. The decision follows a European Medicines Agency (EMA) recommendation in November 2017, which stated the therapy may be of “significant benefit” to Fabry…
Amicus Therapeutics recently submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational oral medicine migalastat for treatment of patients 16 years and older with Fabry disease who have so-called “amenable” mutations. The NDA seeks migalastat’s approval in the U.S. as an alternative to intravenous enzyme…
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