Author Archives: Patricia Inacio PhD

Approval of PRX-102, Plant-based ERT for Adults, Sought for Europe

Protalix BioTherapeutics and its partner Chiesi Global Rare Diseases have applied to the European Medicines Agency (EMA) for the approval of PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy, to treat adults with Fabry disease. This approval request, in the form of a marketing authorization application, has been validated by the EMA and now…

Lucerastat Fails to Lessen Neuropathic Pain in Phase 3 Trial

Lucerastat, an investigational substrate reduction therapy for Fabry disease, has failed to outperform placebo at reducing neuropathic pain in patients, updated results from the MODIFY Phase 3 trial show. The findings mean that Idorsia‘s MODIFY study (NCT03425539) did not meet its primary goal, despite lucerastat being well…

PRX-102 Given to 1st Patient in US Under Expanded Access Program

A first patient has been treated with PRX-102 (pegunigalsidase alfa), an investigational enzyme replacement therapy for Fabry disease that is under review for approval, through an expanded access program (EAP) in the United States. “The initiation of treatment in the first patient enrolled in our EAP is an important milestone for the Fabry…