New monthly dosing of Elfabrio approved in EU for some Fabry patients
Adults with stable disease may seek switch to less frequent infusion schedule
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A monthly dosing regimen for the infusion therapy Elfabrio (pegunigalsidase alfa) has been approved in the European Union for adults with Fabry disease who are stable on enzyme replacement therapy (ERT).
Under the newly cleared regimen, approved by the European Commission, eligible patients may now receive the ERT every four weeks at a dose of 2 mg/kg instead of every-two-week dosing at 1 mg/kg. For those with stable Fabry, this treatment schedule is expected to provide a more convenient dosing alternative.
According to developers Chiesi Global Rare Diseases and Protalix Biotherapeutics, the decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency (EMA) responsible for evaluating new drugs and recommending whether they should be approved in the EU.
Importantly, the older regimen continues to be the only option for patients in the EU who are not stable on ERT. It also remains the only approved dosing regimen in the U.S.
“The European Commission approval for 2mg/kg body weight [every-four-week] dosing regimen for [Elfabrio] represents a meaningful advancement for adults living with Fabry disease and their families,” Giacomo Chiesi, executive vice president at Chiesi Global Rare Diseases, said in a joint company press release announcing the approval.
By extending the frequency of infusions, the new dosing regimen may help reduce treatment burden for eligible patients, their families, and healthcare systems, according to the developers.
“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,” Chiesi said, calling the decision “a milestone” for people with Fabry.
“Ultimately, our goal is simple but profound: to help people spend less time managing their disease and more time living their lives,” Chiesi said.
New dosing option has ‘potential to enhance long-term care’
Fabry disease is caused by mutations that interfere with the production or function of the alpha-galactosidase A (alpha-Gal A) enzyme, which is needed to break down certain fatty molecules, mainly globotriaosylceramide (Gb3), within cells. As a result, these molecules accumulate to toxic levels in and cause damage to organs such as the kidneys, heart, and nervous system, ultimately driving Fabry symptoms.
ERT has been the mainstay Fabry treatment for more than two decades. It works by delivering a functional version of the alpha-Gal A enzyme through regular infusions, helping the body break down Gb3. Such treatments are expected to ease symptoms and potentially slow disease progression.
Available ERTs for Fabry are delivered intravenously, or by infusion into the bloodstream, weekly or every two weeks. However, the enzyme in Elfabrio was designed to be more stable in the blood and remain active for longer, suggesting it had the potential to be given less frequently.
The application seeking the approval of a monthly 2 mg/kg dosing regimen was submitted by the companies and accepted for review by the EMA at the end of 2024.
The submission was in part supported by data from the Phase 3 BRIGHT clinical trial (NCT03180840) and its ongoing open-label extension study (NCT03614234), which evaluated the safety and effectivenss of the less frequent schedule in patients with stable disease. Results from BRIGHT, which enrolled 30 adults with Fabry disease who had been receiving other ERTs, showed that the modified regimen of Elfabrio was generally safe and effective.
Advisory committe had reversed its position on new Elfabrio regimen
The CHMP had previously issued a negative opinion on the proposed modified regimen before ultimately supporting its addition as an alternative for eligible patients.
“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 CEO at Protalix.
Bashan added that “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.”
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.
For patients and advocacy groups, the approval represents a meaningful step toward reducing the day-to-day impact of treatment.
“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, a nonprofit that brings together patient organizations across more than 50 countries.
“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,” Pavlou said.
