Chiesi Global Rare Diseases and Protalix BioTherapeutics Request EMA Re-evaluation of Negative Opinion on Elfabrio (pegunigalsidase alfa) Four-Week Dosing Regimen in the EU
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Nov 03 2025
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Source: Globenewswire
Re-examination Request: Chiesi Global Rare Diseases and Protalix BioTherapeutics have requested a re-examination of the negative opinion from the EMA regarding the proposed dosing regimen of Elfabrio (pegunigalsidase alfa) for Fabry disease, seeking to add a 2 mg/kg every 4 weeks option to the existing 1 mg/kg every 2 weeks regimen.
Safety Information: Elfabrio is associated with hypersensitivity reactions, including anaphylaxis, and patients are advised to have medical support available during administration. Clinical trials reported a significant incidence of infusion-associated reactions among treated patients.
About Fabry Disease: Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leading to serious health complications. Early detection and treatment are crucial for managing symptoms and slowing disease progression.
Chiesi Group Overview: Chiesi is a biopharmaceutical company focused on innovative therapies for rare diseases and is committed to social and environmental responsibility, aiming for net-zero greenhouse gas emissions by 2035.
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About PLX
Protalix BioTherapeutics, Inc. is a biopharmaceutical company. The Company is focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. In addition, the Company is developing PEGylated uricase, or PRX-115, for the treatment of uncontrolled gout, Long Acting (LA) DNase I, or PRX-119, for the treatment of NETs-related diseases, and other technologies and preclinical assets. Its ProCellEx platform is being used to manufacture both of its approved and marketed products as well as PRX-115 and PRX-119. PRX-115, a plant cell-expressed recombinant PEGylated Uricase (urate oxidase) - a chemically modified enzyme to treat uncontrolled gout. PRX-119 is a plant cell-expressed PEGylated recombinant human DNase I product candidate. Its product includes Elelyso and Elfabrio. Elelyso is an enzyme replacement therapy (ERT) for the treatment of patients with Gaucher disease.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Elfabrio's New Dosing Regimen Approved by EU
- EU Dosing Approval: The European Commission has approved a new dosing regimen for Elfabrio (pegunigalsidase alfa) at 2mg/kg every four weeks, aimed at providing more flexible treatment options for stable Fabry disease patients, thereby reducing the treatment burden on patients and their families.
- Burden Reduction: This new regimen extends the infusion interval from every two weeks to every four weeks, helping patients and caregivers better balance long-term treatment with daily life, ultimately enhancing their quality of life.
- Clinical Research Backing: The approval is supported by positive results from the BRIGHT study, demonstrating the new dosing regimen's favorable safety and efficacy profile, further solidifying Elfabrio's position in Fabry disease treatment.
- Significant Market Potential: The collaboration between Chiesi Global Rare Diseases and Protalix BioTherapeutics will drive the widespread adoption of the new dosing regimen across the EU, expected to significantly enhance Elfabrio's market acceptance and sales potential.
See More
Elfabrio's New Dosing Regimen Approved by EU
- EU Dosing Approval: The European Commission has approved a new dosing regimen for Elfabrio (pegunigalsidase alfa) at 2mg/kg every four weeks, aimed at providing greater flexibility for stable Fabry disease patients, thereby reducing the treatment burden on patients and their families.
- Burden Reduction: This new regimen extends the infusion interval from every two weeks to every four weeks, helping patients and families minimize frequent infusion visits, which enhances quality of life and aligns with the long-term treatment needs of Fabry disease patients.
- Clinical Research Support: The approval is backed by positive results from the BRIGHT study, demonstrating the new dosing regimen's favorable safety and efficacy profile, further solidifying Elfabrio's position in the treatment landscape for Fabry disease.
- Market Impact and Collaboration: The partnership between Chiesi Global Rare Diseases and Protalix BioTherapeutics will facilitate the rollout of the new dosing regimen across the EU, expected to expand Elfabrio's market reach and enhance the company's competitiveness in the rare disease sector.
See More
Ellomay Capital Shareholders Sell Stakes and Board Restructuring
- Shareholder Sale: Ellomay Capital's principal shareholders, including S. Nechama Investments, completed the sale of approximately 45.9% of the company's outstanding shares to O.Y. Nofar Energy, potentially leading to significant changes in control and strategic direction for the company.
- Board Restructuring: Following the resignations of two board members and the impending resignation of Chairman Ben Sheizaf, Ellomay's board has been restructured with the appointment of two new independent directors, aimed at introducing fresh perspectives to tackle market challenges.
- New Director Background: Newly appointed director Odelya Ohayon previously served as VP of Marketing at Samsung, managing an annual turnover of NIS 1.5 billion, and is expected to drive market innovation and strategic development at Ellomay.
- Financial Leadership Experience: New director Gilad Mamlok brings 30 years of financial management experience, having served as CFO at Protalix BioTherapeutics, and is anticipated to provide expertise in capital markets transactions and corporate governance, supporting the company's future growth.
See More
Protalix's New Dosing Regimen Recommended for Approval
- New Dosing Regimen Recommendation: The European Medicines Agency committee has recommended approval of Protalix's new dosing regimen for pegunigalsidase alfa (Elfabrio), allowing adult patients to receive treatment every four weeks at a dose of 2mg/kg, significantly reducing patient visits and enhancing treatment convenience.
- Regulatory Milestone Payment: Protalix will be eligible for a $25 million regulatory milestone payment from Chiesi Global Rare Diseases if the new dosing regimen is approved, providing crucial financial support and reducing the risk of future capital raises for the company.
- Positive Market Reaction: Following the announcement, Protalix's stock surged 13%, reflecting investor optimism regarding the new dosing regimen, while sentiment on Stocktwits shifted from 'bullish' to 'extremely bullish', indicating a significant increase in market confidence.
- Future Decision Timeline: The European Medicines Agency is expected to announce a final decision by March 2026, and approval would offer Fabry disease patients a more flexible treatment option, further solidifying Protalix's market position in the rare disease sector.
See More
EMA Endorses New Dosing Regimen for Elfabrio
- New Dosing Regimen Approved: The European Medicines Agency (EMA) expert panel endorsed a new dosing regimen for Elfabrio, recommending a 2 mg/kg every-four-weeks schedule aimed at reducing the treatment burden for patients with Fabry disease.
- Positive Stock Reaction: Following the EMA's favorable opinion, shares of Protalix BioTherapeutics (PLX) surged approximately 14%, reflecting market optimism regarding the new regimen.
- Increased Patient Convenience: The new regimen will reduce infusion frequency for Fabry disease patients from biweekly to once every four weeks, significantly enhancing patient quality of life and treatment adherence.
- Potential Milestone Payment: If the European Commission ultimately approves the regimen, Protalix could receive a regulatory milestone payment of $25 million, further strengthening the company's financial position and market competitiveness.
See More
CHMP Endorses New Treatment Regimen for Fabry Disease
- Positive Opinion Issued: The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the 2mg/kg every-4-weeks dosing regimen for pegunigalsidase alfa after re-examination, with a decision from the European Commission expected by March 2026, which will provide more flexible treatment options for Fabry disease patients.
- Reducing Treatment Burden: If approved, this regimen will significantly alleviate the treatment burden on patients and their families by eliminating the need for bi-weekly visits to infusion centers or home infusions, thereby enhancing patients' quality of life and treatment adherence.
- Clinical Trial Support: The CHMP's positive opinion is informed by results from the BRIGHT study, which assessed the safety and efficacy of pegunigalsidase alfa, indicating that extending infusion intervals allows therapy to better fit into patients' daily lives, supporting work and family commitments.
- Strategic Collaboration Outlook: The partnership between Protalix BioTherapeutics and Chiesi will enable Protalix to receive a $25 million regulatory milestone payment upon approval of the E4W regimen, further advancing its innovation and market expansion in the rare disease sector.
See More
Elfabrio's New Dosing Regimen Approved by EU
- EU Dosing Approval: The European Commission has approved a new dosing regimen for Elfabrio (pegunigalsidase alfa) at 2mg/kg every four weeks, aimed at providing more flexible treatment options for stable Fabry disease patients, thereby reducing the treatment burden on patients and their families.
- Burden Reduction: This new regimen extends the infusion interval from every two weeks to every four weeks, helping patients and caregivers better balance long-term treatment with daily life, ultimately enhancing their quality of life.
- Clinical Research Backing: The approval is supported by positive results from the BRIGHT study, demonstrating the new dosing regimen's favorable safety and efficacy profile, further solidifying Elfabrio's position in Fabry disease treatment.
- Significant Market Potential: The collaboration between Chiesi Global Rare Diseases and Protalix BioTherapeutics will drive the widespread adoption of the new dosing regimen across the EU, expected to significantly enhance Elfabrio's market acceptance and sales potential.
See More
Elfabrio's New Dosing Regimen Approved by EU
- EU Dosing Approval: The European Commission has approved a new dosing regimen for Elfabrio (pegunigalsidase alfa) at 2mg/kg every four weeks, aimed at providing greater flexibility for stable Fabry disease patients, thereby reducing the treatment burden on patients and their families.
- Burden Reduction: This new regimen extends the infusion interval from every two weeks to every four weeks, helping patients and families minimize frequent infusion visits, which enhances quality of life and aligns with the long-term treatment needs of Fabry disease patients.
- Clinical Research Support: The approval is backed by positive results from the BRIGHT study, demonstrating the new dosing regimen's favorable safety and efficacy profile, further solidifying Elfabrio's position in the treatment landscape for Fabry disease.
- Market Impact and Collaboration: The partnership between Chiesi Global Rare Diseases and Protalix BioTherapeutics will facilitate the rollout of the new dosing regimen across the EU, expected to expand Elfabrio's market reach and enhance the company's competitiveness in the rare disease sector.
See More
Ellomay Capital Shareholders Sell Stakes and Board Restructuring
- Shareholder Sale: Ellomay Capital's principal shareholders, including S. Nechama Investments, completed the sale of approximately 45.9% of the company's outstanding shares to O.Y. Nofar Energy, potentially leading to significant changes in control and strategic direction for the company.
- Board Restructuring: Following the resignations of two board members and the impending resignation of Chairman Ben Sheizaf, Ellomay's board has been restructured with the appointment of two new independent directors, aimed at introducing fresh perspectives to tackle market challenges.
- New Director Background: Newly appointed director Odelya Ohayon previously served as VP of Marketing at Samsung, managing an annual turnover of NIS 1.5 billion, and is expected to drive market innovation and strategic development at Ellomay.
- Financial Leadership Experience: New director Gilad Mamlok brings 30 years of financial management experience, having served as CFO at Protalix BioTherapeutics, and is anticipated to provide expertise in capital markets transactions and corporate governance, supporting the company's future growth.
See More
Protalix's New Dosing Regimen Recommended for Approval
- New Dosing Regimen Recommendation: The European Medicines Agency committee has recommended approval of Protalix's new dosing regimen for pegunigalsidase alfa (Elfabrio), allowing adult patients to receive treatment every four weeks at a dose of 2mg/kg, significantly reducing patient visits and enhancing treatment convenience.
- Regulatory Milestone Payment: Protalix will be eligible for a $25 million regulatory milestone payment from Chiesi Global Rare Diseases if the new dosing regimen is approved, providing crucial financial support and reducing the risk of future capital raises for the company.
- Positive Market Reaction: Following the announcement, Protalix's stock surged 13%, reflecting investor optimism regarding the new dosing regimen, while sentiment on Stocktwits shifted from 'bullish' to 'extremely bullish', indicating a significant increase in market confidence.
- Future Decision Timeline: The European Medicines Agency is expected to announce a final decision by March 2026, and approval would offer Fabry disease patients a more flexible treatment option, further solidifying Protalix's market position in the rare disease sector.
See More
EMA Endorses New Dosing Regimen for Elfabrio
- New Dosing Regimen Approved: The European Medicines Agency (EMA) expert panel endorsed a new dosing regimen for Elfabrio, recommending a 2 mg/kg every-four-weeks schedule aimed at reducing the treatment burden for patients with Fabry disease.
- Positive Stock Reaction: Following the EMA's favorable opinion, shares of Protalix BioTherapeutics (PLX) surged approximately 14%, reflecting market optimism regarding the new regimen.
- Increased Patient Convenience: The new regimen will reduce infusion frequency for Fabry disease patients from biweekly to once every four weeks, significantly enhancing patient quality of life and treatment adherence.
- Potential Milestone Payment: If the European Commission ultimately approves the regimen, Protalix could receive a regulatory milestone payment of $25 million, further strengthening the company's financial position and market competitiveness.
See More
CHMP Endorses New Treatment Regimen for Fabry Disease
- Positive Opinion Issued: The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the 2mg/kg every-4-weeks dosing regimen for pegunigalsidase alfa after re-examination, with a decision from the European Commission expected by March 2026, which will provide more flexible treatment options for Fabry disease patients.
- Reducing Treatment Burden: If approved, this regimen will significantly alleviate the treatment burden on patients and their families by eliminating the need for bi-weekly visits to infusion centers or home infusions, thereby enhancing patients' quality of life and treatment adherence.
- Clinical Trial Support: The CHMP's positive opinion is informed by results from the BRIGHT study, which assessed the safety and efficacy of pegunigalsidase alfa, indicating that extending infusion intervals allows therapy to better fit into patients' daily lives, supporting work and family commitments.
- Strategic Collaboration Outlook: The partnership between Protalix BioTherapeutics and Chiesi will enable Protalix to receive a $25 million regulatory milestone payment upon approval of the E4W regimen, further advancing its innovation and market expansion in the rare disease sector.
See More
