Committee for Medicinal Products for Human Use (CHMP) issues a positive opinion following re-examination, which will be reviewed by the European Commission (EC), with a decision anticipated by March 2026 If approved by the EC, this dosing regimen would reduce the burden to eligible adult patients, their families and the broader healthcare system due to the current requirement to visit infusion centres or have home infusions every-2-weeks for treatment PARMA, Italy and CARMIEL, Israel, Jan. 30, 2026 (GLOBE N ...

Committee for Medicinal Products for Human Use (CHMP) issues a positive opinion following re-examination, which will be reviewed by the European Commission (EC), with a decision anticipated by March 2026 If approved by the EC, this dosing regimen would reduce the burden to eligible patients, their families, and the broader healthcare system due to the current requirement to visit infusion centres every two weeks for treatment This dosing regimen for Elfabrio is not approved in the U.S. In the U.S., the appr ...

Core Insights - Chiesi Global Rare Diseases and Protalix BioTherapeutics received a negative opinion from the CHMP of the EMA regarding the approval of a new dosing regimen for Elfabrio (pegunigalsidase alfa) [1][2] - The proposed regimen of 2 mg/kg every 4 weeks was not deemed to have sufficient efficacy compared to the currently approved regimen of 1 mg/kg every 2 weeks [1][2] Company Overview - Chiesi Global Rare Diseases is a unit of the Chiesi Group focused on innovative therapies for rare diseases [1][16] - Protalix BioTherapeutics specializes in developing recombinant therapeutic proteins using its proprietary ProCellEx® plant cell-based expression system [1][19] - Both companies are committed to reducing the treatment burden for patients with Fabry disease [2][20] Clinical Trial Insights - The CHMP review was based on data from the BRIGHT trial and its extension study, which had a median exposure of almost six years [2] - The data from these studies, along with modeling and exposure-response analyses from prior trials, were insufficient to demonstrate similar efficacy for the new dosing regimen [2] Fabry Disease Context - Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, leading to serious health complications [11][12] - Early detection and access to treatment are critical for managing symptoms and slowing disease progression [12] Safety Information - Elfabrio is indicated for adults with confirmed Fabry disease and has been associated with hypersensitivity reactions, including anaphylaxis [3][4] - In clinical trials, 14% of patients experienced hypersensitivity reactions, with 3% experiencing anaphylaxis [6][9]