This press release is intended for US audiences for transparency relative to global news for the Fabry community. This dosing regimen for Elfabrio is not approved in the US. In the US, the FDA-approved dosing regimen remains 1mg/kg every 2 weeks. Please see Important Safety Information below and the Full Prescribing Information, including Boxed Warning.
European Commission approved dosing regimen reduces the burden for eligible patients, their families, and the broader healthcare system by extending infusion interval frequency from every-two-weeks to every-four-weeks for those stable with an enzyme replacement therapy (ERT)
With this decision, announced ahead of Fabry Disease Awareness Month in April, Chiesi Global Rare Diseases will work with countries across the EU to support broader access to this additional dosing schedule for the adult Fabry community
Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, and Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the discovery, development, production and commercialization of innovative therapeutics for rare diseases with significant unmet needs, today announced that the European Commission (EC) has approved the 2mg/kg every-4-weeks (E4W) dosing regimen for Elfabrio® (pegunigalsidase alfa) in adults living with Fabry disease who are stable with an ERT. The EC decision follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the additional dosing regimen.
“The European Commission approval for 2mg/kg body weight E4W dosing regimen for pegunigalsidase alfa represents a meaningful advancement for adults living with Fabry disease and their families,” said Giacomo Chiesi, Executive Vice President, Chiesi Global Rare Diseases. “Because Fabry disease requires lifelong treatment, the cadence of therapy inevitably becomes part of everyday life for patients and caregivers. By introducing an option that extends the infusion interval from every two weeks to every four weeks for eligible patients on stable ERT, we are offering families greater flexibility and the possibility to ease the overall burden of treatment. Ultimately, our goal is simple but profound: to help people spend less time managing their disease and more time living their lives. This milestone reflects our commitment to innovation that goes beyond delivering therapies—by listening to and understanding the real experiences of the Fabry community.”
“In Fabry disease, long-term treatment decisions must balance disease management with the realities of lifelong therapy,” said Prof. Aleš Linhart, DrSc, FESC. “The approval of pegunigalsidase alfa 2mg/kg every-4-weeks provides an additional option that may help reduce cumulative treatment burden for appropriate patients while maintaining continuity of care.”
“This approval strengthens the treatment landscape for Fabry disease across the European Union by introducing an additional dosing approach that has the potential to enhance long-term care,” said Dror Bashan, President and Chief Executive Officer, Protalix BioTherapeutics. “The authorization reflects not only scientific progress, but also a commitment to optimizing care delivery in a way that supports both patients and healthcare systems.”
“For many people living with Fabry disease, treatment is a lifelong commitment that impacts nearly every aspect of daily life,” said Mary Pavlou, President, Fabry International Network (FIN). “This approval allows for fewer infusion visits, helping reduce the ongoing burden on patients and families, allowing them to spend more time living their lives beyond treatment.”
The EC approval is informed by results from an open-label, switch-over study, BRIGHT (formally PB-102-F50), designed to assess the adverse-event profile, efficacy, and pharmacokinetics (PK) of the alternative dosing regimen of pegunigalsidase alfa 2-mg/kg E4W for 52 weeks,1 and its ongoing open-label extension study CLI-06657AA1-03 (formerly PB-102-F51).2
Protalix is entitled to a regulatory milestone payment of $25 million from Chiesi in connection with the EC’s approval of the E4W dosing regimen.
This EU approval does not change the FDA‑approved dosing regimen, which remains 1-mg/kg every 2 weeks. Please consult with your healthcare provider.
