Protalix Biotherapeutics Inc (NYSE-A:PLX, FRA:PBDA) announced that the European Commission has approved a new dosing regimen for pegunigalsidase alfa for adults with Fabry disease who are stable on enzyme replacement therapy (ERT), triggering a $25 million regulatory milestone payment from partner Chiesi Global Rare Diseases.
The approval allows eligible patients to receive the therapy at a dose of 2 mg/kg every four weeks, instead of the previous every-two-weeks schedule. According to the companies, the change could reduce the treatment burden for patients, their families and healthcare systems by extending the interval between infusions.
The decision follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), which recommended the additional dosing regimen.
The approval was supported by results from the open-label BRIGHT study, which evaluated the safety, efficacy and pharmacokinetics of the every-four-weeks regimen over 52 weeks, as well as data from an ongoing open-label extension study.
“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,” Protalix CEO Dror Bashan said in a statement.
“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 Global Rare Diseases said it will work with countries across the European Union to support broader access to the new dosing schedule for the adult Fabry community. The announcement comes ahead of Fabry Disease Awareness Month in April.
“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,” Chiesi Global Rare Diseases president Giacomo Chiesi said.
“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.”
Shares of Protalix were up 6.5% on Monday morning.