Street Calls of the Week
PARMA/CARMIEL - Chiesi Global Rare Diseases and Protalix BioTherapeutics (NYSE American:PLX), a biotech company that has seen its stock surge 120% over the past year, announced Friday that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on their application to approve an additional dosing regimen for Elfabrio. According to InvestingPro data, Protalix maintains a strong financial health score of "GREAT," with robust cash reserves exceeding its debt obligations.
The companies had sought approval for a 2 mg/kg body weight infusion every four weeks (E4W) for pegunigalsidase alfa, in addition to the currently approved 1 mg/kg every two weeks (E2W) regimen for treating Fabry disease.
The submission was based on data from the BRIGHT trial and its extension study, which together provided nearly six years of median exposure data. Additional support came from modeling and exposure-response analyses across multiple trials. However, the CHMP determined these data were insufficient to conclude similar efficacy for the proposed dosing schedule.
"We are disappointed by the result of this review but want to express our immense appreciation for the collaboration of the patient community, researchers and European Commission throughout this process," said Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases.
Dror Bashan, Protalix’s President and CEO, stated that both companies remain committed to reducing treatment burden for Fabry disease patients despite this setback.
Elfabrio was approved by both the FDA and European Medicines Agency in May 2023 for treating adults with confirmed Fabry disease. The medication carries a boxed warning for hypersensitivity reactions, including anaphylaxis.
Fabry disease is a rare inherited disorder caused by mutations in the GLA gene, leading to enzyme deficiency that affects multiple organs including the heart, kidneys, and nervous system.
The companies indicated they will continue working together to support the Fabry disease community. This announcement was based on a company press release statement.
In other recent news, Protalix BioTherapeutics reported its second-quarter 2025 earnings, showcasing a mixed financial performance. The company missed earnings per share (EPS) expectations, reporting -$0.04 against the anticipated $0.11, a significant miss of 136.36%. However, Protalix exceeded revenue expectations by reporting $25.77 million, surpassing the forecasted $23.10 million by 11.56%. Additionally, Protalix amended its at-the-market equity offering agreement with H.C. Wainwright & Co., LLC. This amendment updates the sales agreement to align with the company’s new registration statement on Form S-3. Under this agreement, Protalix may continue to offer and sell shares of its common stock through H.C. Wainwright & Co. Protalix also filed a legal opinion from Mayer Brown LLP regarding the validity of the shares to be issued under the program. These developments reflect ongoing strategic financial adjustments within the company.
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