The European Medicines Agency (EMA) has validated Chiesi Global Rare Diseases and Protalix BioTherapeutics’ submission to update the dosing regimen of pegunigalsidase alfa, used in treating Fabry disease, a rare genetic lysosomal storage disorder.
The proposed regimen suggests a dose of 2 mg/kg body weight administered every four weeks for adults, a modification from the currently approved bi-weekly 1 mg/kg dose.
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Chiesi and Protalix’s variation application for pegunigalsidase alfa’s new dosing regimen is backed by a revised population-PK model, new exposure-response analyses and clinical data from the completed Phase III BRIGHT trial and its ongoing extension study.
The studies explored the 2mg/kg every four weeks dosing for adults previously treated with agalsidase-alfa or beta.
Chiesi Global Rare Diseases executive vice-president Giacomo Chiesi said: “The validation of this variation application is an important milestone in our efforts to reduce the burden of treatment for some adult patients living with Fabry disease who continue to experience unmet medical needs.
“We are committed to delivering innovative therapies and solutions for people living with Fabry disease, their families and caregivers.”
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By GlobalDataA biopharmaceutical company, Protalix leverages its plant cell-based expression system, ProCellEx, to develop recombinant therapeutic proteins.
Pegunigalsidase alfa, the company’s second product, received approval from both the US Food and Drug Administration (FDA) and the EMA in May 2023.
In the same month, the European Commission granted marketing authorisation to the therapy to treat the condition.
Protalix BioTherapeutics CEO and president Dror Bashan stated: “Based on study results, we believe in the potential of pegunigalsidase alfa 2 mg/kg administered every four weeks to be a beneficial alternative dosing option for some adults living with Fabry disease.
“Together with Chiesi, we remain committed to meeting the needs of people with Fabry disease and bringing additional therapeutic options to market. We look forward to continuing to work closely with the agency in the months ahead.”
The partnership between Chiesi Farmaceutici and Protalix focuses on the development and commercialisation of pegunigalsidase alfa worldwide.
Chiesi Global Rare Diseases is a business division of the Chiesi Group.
