EU Approves Monthly Elfabrio Dose, Halving Fabry Patient Burden

PARMA, Italy & CARMIEL, Israel – March 09, 2026 – The European Commission (EC) has granted a significant approval that promises to reshape the lives of adults living with Fabry disease, a rare and debilitating genetic disorder. Chiesi Global Rare Diseases and Protalix BioTherapeutics announced today that their enzyme replacement therapy, Elfabrio® (pegunigalsidase alfa), is now approved for an every-four-weeks (E4W) dosing regimen for eligible patients.

This decision, which follows a positive recommendation from the European Medicines Agency’s (EMA) scientific committee, allows adults who are stable on an existing enzyme replacement therapy (ERT) to extend their infusion intervals from every two weeks to once a month. The move is being hailed as a major advancement in patient-centric care, aimed at reducing the immense logistical and emotional burden associated with lifelong treatment.

A New Horizon for Patient Quality of Life

Fabry disease is an inherited lysosomal storage disorder where a deficient enzyme leads to the harmful buildup of a fatty substance, GL-3, in cells throughout the body. This accumulation can cause severe, progressive damage to the heart, kidneys, and nervous system, resulting in chronic pain, fatigue, and life-threatening complications. For decades, the standard of care has been ERT, which typically requires intravenous infusions every two weeks for a patient's entire life.

“For many people living with Fabry disease, treatment is a lifelong commitment that impacts nearly every aspect of daily life,” said Mary Pavlou, President of the 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 shift to a monthly schedule represents more than just a change in frequency; it signifies a profound improvement in quality of life. Patients can reclaim dozens of hours previously spent traveling to and from clinics, undergoing infusions, and recovering. This newfound flexibility can translate into more consistent employment, uninterrupted school attendance, and the simple freedom to plan life more than two weeks at a time. The psychological relief from a less intrusive treatment schedule is a critical, though less quantifiable, benefit.

The EC’s decision is backed by solid clinical evidence from the BRIGHT study. This open-label trial was specifically designed to assess the safety and efficacy of switching stable patients to a 2 mg/kg E4W dosing regimen of Elfabrio. The results, supported by an ongoing extension study, demonstrated that the monthly dose maintained therapeutic effectiveness while being well-tolerated, providing the regulatory confidence needed for this landmark approval.

Reshaping a Competitive Therapeutic Landscape

The approval strategically positions Elfabrio® in Europe's competitive Fabry disease market. For years, the space has been dominated by established bi-weekly ERTs like Sanofi’s Fabrazyme® and Takeda’s Replagal®. While effective, the frequent dosing schedule of these therapies has remained a significant drawback for patients. By offering a clinically validated monthly option, Chiesi and Protalix have introduced a powerful differentiator that directly addresses a major unmet need for convenience and reduced treatment burden.

“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 of Chiesi Global Rare Diseases. “Ultimately, our goal is simple but profound: to help people spend less time managing their disease and more time living their lives.”

The therapy also competes in a landscape that includes oral options like Amicus Therapeutics' Galafold®, a chaperone therapy. However, Galafold is only suitable for patients with specific genetic mutations. Elfabrio’s broader applicability as an ERT, now coupled with a more convenient dosing schedule, allows it to carve out a unique and compelling value proposition for a wider segment of the Fabry patient population.

“In Fabry disease, long-term treatment decisions must balance disease management with the realities of lifelong therapy,” noted 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.”

Strategic Gains and System-Wide Benefits

Beyond the patient and competitive implications, this approval triggers significant strategic and financial developments. For Protalix BioTherapeutics, the Israeli biopharmaceutical firm that developed Elfabrio using its proprietary ProCellEx® plant cell-based expression system, the EC’s decision triggers a $25 million milestone payment from its partner, Chiesi. This non-dilutive cash infusion provides a substantial boost to Protalix’s finances, strengthening its ability to advance its R&D pipeline, which includes promising candidates for gout (PRX-115) and other rare diseases.

For European healthcare systems, the E4W regimen offers a path toward greater efficiency and resource optimization. Halving the number of annual infusions per patient could lead to significant downstream benefits, including reduced demand for infusion center chairs, less time spent by specialized nursing staff on administration, and streamlined logistics. These efficiencies can free up critical resources to be allocated to other areas of patient 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 of 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.”

Chiesi’s Deepening Commitment to Rare Diseases

This regulatory success is a cornerstone of Chiesi Global Rare Diseases' ambitious strategy. Established in 2020, the unit has rapidly become a significant growth driver for the parent Chiesi Group, with its revenue climbing to €763 million in 2024. The approval of the E4W regimen for Elfabrio perfectly embodies the unit’s mission to deliver patient-centric innovation that goes beyond the molecule itself to address the holistic experience of living with a rare disease.

Chiesi has aggressively expanded its footprint in the sector through both strategic partnerships, like the one with Protalix, and major acquisitions, such as its 2023 purchase of Amryt Pharma. This has been supported by a massive commitment to research and development, with R&D spending reaching a record 24.3% of the company’s revenue in 2024.

With this latest approval, Chiesi not only strengthens its product portfolio but also reinforces its reputation as a company that listens to the rare disease community. As Chiesi works to roll out the new dosing schedule across EU member states, the focus will be on ensuring that this significant therapeutic advancement translates into tangible, life-changing benefits for the thousands of individuals and families affected by Fabry disease.