News

A small fat molecule that accumulates in the tissues of Fabry disease patients may be used as a biomarker for early diagnosis in the absence of recognizable clinical symptoms, according to a case report. Globotriaosylsphingosine, or lyso-Gb3, is a fat molecule that is broke down by an enzyme called…

The U.S. Food and Drug Administration will be giving priority review to Amicus Therapeutics’ Fabry disease therapy Galafold (migalastat). Fabry disease is caused by mutations of the GLA gene. Amicus asked the FDA to approve Galafold for Fabry patients older than 16 with mutations that can respond to migalastat. Up to half…

AVROBIO has obtained $60 million in additional financing to start a Phase 2 clinical trial of its gene therapy for Fabry disease. The second round of financing will also help the company continue developing treatments for other lysosomal storage disorders, Gaucher disease, cystinosis, and Pompe disease. A Phase 1 trial (…

Amicus Therapeutics recently announced the launch of Galafold (migalastat) in Spain for the long-term treatment of anyone who has Fabry disease and is older than 16 years. Following final regulatory adjustments, Galafold is now reimbursed in Spain for age-eligible patients with a confirmed diagnosis of Fabry disease and who have…

The Canadian Drug Expert Committee (CDEC) has recommended in favor or reimbursement of Galafold (migalastat) for long-term treatment of adults with a confirmed diagnosis of Fabry disease and who have an amenable mutation, Amicus Therapeutics announced. CDEC will now start conversations with regulatory authorities to make Galafold available to Canadian…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review of investigational drugs and vaccines for serious conditions…

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…