Protalix Biotherapeutics Inc (NYSE-A:PLX, FRA:PBDA) said on Friday that the European Medicines Agency’s human medicines committee has issued a positive opinion recommending approval of an expanded dosing regimen for Elfabrio, its treatment for Fabry disease, in adult patients.
The Committee for Medicinal Products for Human Use (CHMP) recommended approval of a 2 mg/kg every-four-weeks dosing schedule for Elfabrio in adult Fabry patients who are stable on enzyme replacement therapy, following a re-examination of the application.
Elfabrio, also known as pegunigalsidase alfa, is commercialized in partnership with Chiesi Global Rare Diseases, a unit of Italy’s Chiesi Group, which focuses on therapies for rare conditions.
Protalix said the CHMP opinion was based on results from the BRIGHT open-label switch-over 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 with a median patient exposure of nearly six years. Additional support came from updated population pharmacokinetics and exposure-response analyses using data from multiple clinical studies.
“The CHMP’s positive opinion is another testament to Protalix’s commitment to advancing treatments for people living with Fabry disease,” Protalix CEO Dror Bashan said, adding that the decision validates the company’s development pipeline and its proprietary ProCellEx manufacturing platform.
Chiesi Global Rare Diseases executive vice president Giacomo Chiesi said the less frequent dosing regimen could help reduce treatment burden for patients by extending the time between infusions.
Approval of the new dosing schedule by the European Commission would trigger a $25 million regulatory milestone payment to Protalix from Chiesi, the company said.
Fabry disease is a rare, inherited metabolic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to progressive damage to organs, including the heart and kidneys.
Shares of Protalix gained 8.7% on the news Friday morning in New York.