Protalix BioTherapeutics Inc (AMEX:PLX) shares tumbled 20.8% in premarket trading on Friday after the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the company’s application to approve an alternative dosing schedule for its Fabry disease therapy Elfabrio.
The CHMP rejected Protalix’s proposal for a 2 mg/kg body weight infusion every four weeks (E4W) as an alternative to the currently approved 1 mg/kg every two weeks (E2W).
The submission was supported by data from the BRIGHT trial — an open-label, switch-over study — and its ongoing extension study, with a median patient exposure of nearly six years. Additional evidence came from modeling and exposure–response analyses from previous trials. However, the CHMP concluded that the evidence was “not deemed sufficient to conclude on similar efficacy.”
Despite the regulatory setback, Protalix and its partner Chiesi Global Rare Diseases reiterated their commitment to easing the treatment burden for Fabry disease patients. Elfabrio is a recombinant therapeutic protein produced using Protalix’s proprietary ProCellEx plant cell-based expression system.
“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.
Dror Bashan, President and CEO of Protalix, added that the companies remain focused on “reducing treatment burden for Fabry disease patients despite this regulatory outcome.”
Both firms stated they will continue to work together to support the Fabry disease community going forward.
Protalix BioTherapeutics stock price
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