Protalix Biotherapeutics

In 2024, Fabry Disease News highlighted key updates: gene therapy trials for Fabry disease showed promise, including AMT-191 and 4D-310 targeting heart and liver. Elfabrio slowed kidney decline. Studies found elevated inflammatory markers in patients and suggested small nerve fiber damage as a diagnostic aid. An algorithm using insurance data aims to speed up diagnosis. Chronic cough and pain were identified as potential Fabry symptoms.

Protalix BioTherapeutics, Inc. announced significant milestones in 2024, including EMA validation for a less frequent dosing regimen of pegunigalsidase alfa for Fabry disease, progress in PRX-115 clinical trials for uncontrolled gout, and repayment of all outstanding convertible notes, positioning the company for long-term growth.

The EMA validated an application for a less frequent Elfabrio regimen (2 mg/kg every 4 weeks) for Fabry disease, based on Phase 3 BRIGHT trial data showing its safety and effectiveness. This could reduce treatment burden for some patients.

EMA validated Chiesi and Protalix’s submission to update pegunigalsidase alfa dosing for Fabry disease to 2 mg/kg every four weeks, supported by clinical data and analyses.

EMA validated Chiesi and Protalix's submission to update pegunigalsidase alfa's dosing regimen for Fabry disease, proposing a 2 mg/kg dose every four weeks. The application is supported by clinical data and analyses, aiming to reduce treatment burden for adult patients.