Protalix BioTherapeutics and Chiesi Global Rare Diseases have requested a re‑examination of the European Medicines Agency’s negative opinion on a proposed 2 mg/kg every‑four‑weeks dosing schedule for Elfabrio, the PEGylated enzyme replacement therapy approved for Fabry disease in May 2023.
The request follows the EMA Committee for Medicinal Products for Human Use (CHMP) decision that the data submitted for the 2 mg/kg every‑four‑weeks regimen were insufficient to demonstrate comparable efficacy to the currently approved 1 mg/kg every‑two‑weeks schedule. The companies argue that the Phase 3 BRIGHT trial and its extension provide evidence of similar safety and efficacy with reduced infusion frequency, potentially improving patient adherence and quality of life.
Protalix reported a 75 % year‑over‑year revenue increase in Q3 2024 and a 16 % rise in Q2 2025, driven largely by sales to Chiesi. The company remains debt‑free as of September 30 2024 and has transitioned to net income. The re‑examination could strengthen Elfabrio’s market position in a Fabry disease market projected to reach USD 4.87 billion by 2034.
Elfabrio carries a boxed warning for hypersensitivity reactions, with 14 % of trial participants experiencing such reactions and 3 % anaphylaxis. The companies emphasize that the safety profile remains consistent across dosing schedules. Management stated that the re‑examination is a critical step toward expanding treatment options for patients and improving access to the therapy.
The existing marketing authorization for Elfabrio remains in force while the EMA review proceeds. Stakeholders will monitor the outcome of the re‑examination, which could influence future sales volumes and pricing negotiations with payers.
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