The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a negative opinion for Protalix BioTherapeutics, Inc.’s (NYSE:PLX) and Chiesi Group’s partnered Elfabrio’s revised dosing schedule for Fabry disease.
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Fabry disease is a rare genetic lysosomal storage disorder in which the body lacks sufficient amounts of an enzyme that breaks down fats.
The companies requested to approve the dosing regimen of 2 mg/kg body weight infused every 4 weeks (E4W) for Elfabrio (pegunigalsidase alfa, in addition to the currently approved dosing regimen of 1 mg/kg body weight infused every 2 weeks (E2W).
“We are disappointed by the result of this review…” said Giacomo Chiesi, Executive Vice President, Chiesi Global Rare Diseases.”
The submission for CHMP review was based on data from the BRIGHT trial (formally PB-102-F50) of pegunigalsidase alfa 2 mg/kg administered every four weeks, as well as its ongoing open-label extension study, CLI-06657AA1-03 (formerly PB-102-F51).
The two studies combined have a median exposure of almost six years.
Further support was provided from modeling and exposure-response analyses across prior trials (PB-102-F01/-F02, PB-102-F20, and PB-102-F50).
These data were not deemed sufficient to conclude on similar efficacy.
In September, uniQure N.V. (NASDAQ:QURE) shared initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.
As of the July 24, 2025, study cutoff date, all four patients in the first cohort, Cohort A (6×1013 genome copies/kilogram (gc/kg)), showed substantial increases in α-Gal A activity, ranging from 27- to 208-fold above the mean normal level.
Price Action: PLX stock is down 29.83% at $1.68 at the last check on Friday.
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