Chiesi Global Rare Diseases and Protalix BioTherapeutics have received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending approval of a new every-four-week dosing regimen of Elfabrio for adult patients with Fabry disease who are stable on enzyme replacement therapy. The opinion follows a re-examination of the application and will now be reviewed by the European Commission, with a decision expected by March 2026. If approved, this regimen would reduce the frequency of infusion visits from every two weeks to every four weeks, easing the burden for patients, families, and the healthcare system.
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Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases, said that the positive opinion represents a meaningful step forward in reducing treatment burden for people living with Fabry disease. He emphasized that the company aims to deliver therapies that not only treat the condition effectively but also integrate smoothly into patients’ daily lives, allowing them to focus on what matters most.
Prof. Aleš Linhart highlighted the importance of offering flexible treatment options that align with patient preferences and minimize disruptions to work, study, and family life. Dror Bashan, President and CEO of Protalix BioTherapeutics, added that the CHMP opinion validates the company’s innovative pipeline and the proprietary ProCellEx manufacturing platform, while acknowledging the contribution of patients, investigators, and clinical staff who participated in the studies.
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Mary Pavlou, President of the Fabry International Network, noted that extending infusion intervals allows patients to better manage their daily routines and maintain quality of life. She described the decision as a recognition of the need for treatments that fit into real-world lifestyles.
The CHMP opinion is based on data from the BRIGHT study, an open-label, switch-over trial assessing the safety, efficacy, and pharmacokinetics of the 2 mg/kg every-four-week regimen for 52 weeks, along with data from the ongoing extension study CLI-06657AA1-03, which includes a median exposure of nearly six years. Additional support comes from updated population pharmacokinetics modeling and exposure–response analyses across multiple clinical studies.
If approved by the European Commission, Protalix would receive a $25 million regulatory milestone payment from Chiesi. This dosing regimen is currently not approved in the United States, where the recommended dose remains 1 mg/kg every two weeks. Patients are advised to consult their healthcare providers regarding treatment options.
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