Protalix Biotherapeutics Inc (NYSE-A:PLX) and Chiesi Global Rare Diseases said they have requested a re-examination of a recent negative opinion by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) concerning a new dosing regimen for Elfabrio.
The CHMP’s opinion relates to a proposal to add a 2 mg/kg every-four-weeks (E4W) dosing option to the currently approved 1 mg/kg every-two-weeks (E2W) regimen for the treatment of adults with Fabry disease.
Both companies said in a joint statement they remain “fully committed to working closely with the EMA” throughout the re-examination process and to addressing the unmet medical needs of the Fabry community.
The existing marketing authorization for Elfabrio remains valid while the re-examination is under way and until a final decision is issued by the European Commission, the companies said.
Elfabrio, also known as pegunigalsidase alfa-iwxj, is approved for use in adults with confirmed Fabry disease, a rare genetic disorder caused by the buildup of a type of fat in the body’s cells.