Chiesi and Protalix BioTherapeutics receive negative CHMP opinion for Elfabrio dosing regimen.

Chiesi Global Rare Diseases and Protalix BioTherapeutics acknowledge that the European Medicines Agency's Committee for Medicinal Products for Human Use has issued a negative opinion on the request to approve the dosing regimen of 2 mg/kg body weight infused every 4 weeks for Elfabrio (pegunigalsidase alfa). The company will continue to collaborate with the agency to address the concerns and ultimately approve the treatment.

Chiesi Global Rare Diseases and Protalix BioTherapeutics have acknowledged that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion on the request to approve the dosing regimen of 2 mg/kg body weight infused every 4 weeks (E4W) for Elfabrio (pegunigalsidase alfa). The current approved dosing regimen for Elfabrio is 1 mg/kg body weight infused every 2 weeks (E2W).

The decision was based on data from an open-label, switch-over trial, BRIGHT (formally PB-102-F50), and its ongoing open-label extension study, CLI-06657AA1-03 (formerly PB-102-F51). These studies combined have a median exposure of almost six years. Additional support was provided from modeling and exposure-response analyses across prior trials (PB-102-F01/-F02, PB-102-F20, and PB-102-F50). The data were not deemed sufficient to conclude on similar efficacy compared to the E2W regimen.

Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases, expressed disappointment with the result but appreciated the collaboration of the patient community, researchers, and European Commission throughout the process. Dror Bashan, Protalix's President and Chief Executive Officer, reaffirmed the commitment to reducing the treatment burden for patients with Fabry disease. Mary Pavlou, President of Fabry International Network (FIN), acknowledged the outcome with gratitude for the dedication shown by all involved.

Chiesi and Protalix intend to continue working together to support the Fabry disease community. The companies will collaborate with the EMA to address the concerns and ultimately seek approval for the E4W dosing regimen.