Chiesi Global Rare Diseases and Protalix BioTherapeutics Seek Re-examination from the EMA for the Negative Opinion for Elfabrio (pegunigalsidase alfa) Alternative Dosing Regimen of Every Four Weeks in the EU

Core Viewpoint - Chiesi Global Rare Diseases and Protalix BioTherapeutics have requested a re-examination of the negative opinion from the CHMP regarding the proposed dosing regimen for Elfabrio® (pegunigalsidase alfa) [1][2] Company Overview - Chiesi Global Rare Diseases is a business unit of the Chiesi Group, focused on delivering innovative therapies for rare diseases [16] - Protalix BioTherapeutics specializes in developing recombinant therapeutic proteins using its proprietary plant cell-based expression system, ProCellEx [18] Product Information - Elfabrio® is indicated for the treatment of adults with confirmed Fabry disease [3] - The current approved dosing regimen is 1 mg/kg every 2 weeks, while a new regimen of 2 mg/kg every 4 weeks is under review [2] Clinical Trial Insights - In clinical trials, 14% of patients treated with Elfabrio experienced hypersensitivity reactions, with 3% experiencing anaphylaxis [6] - Infusion-associated reactions were reported in 29% of patients, including symptoms such as nausea, chills, and rash [7][9] Regulatory Context - The existing marketing authorization for Elfabrio remains effective while the re-examination process is ongoing [2] - The outcome of the re-examination will influence the final decision by the European Commission [2] Disease Background - Fabry disease is a rare inherited lysosomal storage disorder caused by mutations in the GLA gene, leading to enzyme deficiency and accumulation of globotriaosylceramide (GL-3) [11][12] - Symptoms include fatigue, chronic pain, and increased risk of serious complications such as kidney failure and stroke [11][12]