Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for an Additional Dosing Regimen of Every Four Weeks for Elfabrio® (pegunigalsidase alfa) in the EU

Core Viewpoint - The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the approval of a new dosing regimen for Elfabrio (pegunigalsidase alfa) for the treatment of Fabry disease, which, if approved by the European Commission, would significantly reduce the treatment burden for patients and their families [1][2]. Company Updates - Chiesi Global Rare Diseases and Protalix BioTherapeutics announced the positive CHMP opinion for the 2 mg/kg every-four-weeks (E4W) dosing regimen for Elfabrio, aimed at adult patients with Fabry disease who are stable on enzyme replacement therapy [1][2]. - Protalix is set to receive a regulatory milestone payment of $25 million from Chiesi if the E4W dosing regimen is approved by the European Commission [3]. Product Information - Elfabrio is a PEGylated enzyme replacement therapy designed to treat Fabry disease, utilizing a plant cell culture-expressed and chemically modified version of the α-Galactosidase-A enzyme [4]. - Clinical studies have shown that Elfabrio has an initial half-life of 78.9 ± 10.3 hours, indicating stable pharmacokinetic parameters [4]. Clinical Study Insights - The CHMP's positive opinion is based on results from the BRIGHT study, which assessed the safety, efficacy, and pharmacokinetics of the new dosing regimen over 52 weeks, along with an ongoing open-label extension study [2]. - The updated Population Pharmacokinetics model and exposure–response analysis further support the new dosing regimen, leveraging data from multiple clinical studies [2]. Industry Context - The approval of the new dosing regimen is seen as a critical step in expanding treatment options for patients with Fabry disease, addressing both effective management of the condition and patient preferences to minimize disruptions in daily life [2]. - The collaboration between Chiesi and Protalix highlights the commitment to advancing treatments for rare diseases, with a focus on innovative therapeutic solutions [2][19].