Protalix, Chiesi Reaffirm Commitment to Fabry Disease Patients Despite EMA Panel Setback

Protalix Biotherapeutics and Chiesi Global Rare Diseases have reaffirmed their commitment to advancing treatment options for Fabry disease despite a setback from the European Medicines Agency. The agency declined to recommend approval of a new dosing regimen for Elfabrio (pegunigalsidase alfa), citing insufficient data on its efficacy, according to a TradingView report.

Protalix BioTherapeutics and Chiesi Global Rare Diseases have reaffirmed their commitment to advancing treatment options for Fabry disease despite a recent setback from the European Medicines Agency (EMA). The EMA declined to recommend approval of a new dosing regimen for Elfabrio (pegunigalsidase alfa) due to insufficient data on its efficacy, according to a TradingView report.

On October 17, 2025, the EMA rejected the 4-week dosing regimen for Elfabrio, maintaining the 2-week regimen. The decision was based on insufficient data from clinical trials and analyses, as noted in the TradingView report. Despite this setback, both companies remain dedicated to reducing treatment burdens for patients with Fabry disease and continue to support the community, according to a GlobeNewswire statement.

The submission for CHMP review was based on data from an open-label, switch-over trial, BRIGHT, and its ongoing open-label extension study, CLI-06657AA1-03. These studies, along with prior trials, provided data that was not deemed sufficient to conclude on similar efficacy, and Chiesi and Protalix intend to continue working together to support the Fabry disease community and advance treatment options.

Fabry disease is a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene, leading to a deficiency of the enzyme alpha-galactosidase A. This deficiency results in an accumulation of a fatty substance called globotriaosylceramide (GL-3) in the body’s cells, affecting the heart, kidneys, skin, nervous system, and other organs. The condition can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke, as described in the companies' announcement.

Protalix, with its proprietary plant cell-based expression system, ProCellEx, and Chiesi, with its extensive research and development capabilities, are well-positioned to continue advancing treatment options for Fabry disease. Despite the recent setback, the companies' commitment to improving patient outcomes remains steadfast.