Chiesi Global Rare Diseases and Protalix BioTherapeutics announced that the European Medicines Agency (EMA) validated a Variation Submission for pegunigalsidase alfa, marketed as Elfabrio. This submission seeks approval for a less frequent dosing regimen of 2 mg/kg body weight administered every four weeks for adult patients with Fabry disease. The currently approved dose is 1 mg/kg administered every two weeks.
The variation application is supported by a revised Population-PK model, new exposure-response analyses, and clinical data from the completed Phase 3 study PB-102-F50 (BRIGHT) and its ongoing extension study CLI-06657AA1-03. These studies investigated the 2 mg/kg every four weeks dosing regimen in adult Fabry disease patients previously treated with agalsidase-alfa or -beta.
This validation is a significant step in potentially reducing the treatment burden for some adult patients living with Fabry disease. A less frequent dosing regimen could enhance patient compliance and treatment flexibility, offering a differentiated option in the Fabry disease market. Protalix and Chiesi are committed to bringing additional therapeutic options to market.
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