Protalix Biotherapeutics Inc (NYSE-A:PLX) and Chiesi Global Rare Diseases said they remain committed to advancing treatment options for Fabry disease after a European Medicines Agency committee declined to recommend approval of a new, less frequent dosing regimen for Elfabrio (pegunigalsidase alfa).
The Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the proposed dosing schedule of 2 mg/kg every four weeks, in addition to the already approved 1 mg/kg every two weeks regimen.
Protalix CEO Dror Bashan emphasized that the company’s focus remains unchanged. “We, together with Chiesi, remain committed to reducing the treatment burden for patients with Fabry disease,” he said.
“We are disappointed by the result of this review but want to express our immense appreciation for the collaboration of the patient community, researchers and European Commission throughout this process,” said Giacomo Chiesi, executive vice president of Chiesi Global Rare Diseases.
The review was based on data from the BRIGHT open-label trial and its extension study, with a median patient exposure of nearly six years.
Although the data were not deemed sufficient to demonstrate equivalent efficacy for the monthly dosing regimen, both companies said they will continue to collaborate on research and support efforts to advance Fabry disease care.