Chiesi and Protalix Achieve Milestone in Fabry Disease Treatment with Pegunigalsidase Alfa
Chiesi Global Rare Diseases, part of the innovative Chiesi Group, in collaboration with Protalix BioTherapeutics, has made significant progress in the treatment of Fabry disease. The European Medicines Agency (EMA) has validated a submission for a variation, allowing a less frequent dosing regimen for pegunigalsidase alfa, a therapeutic protein crucial in managing this rare genetic disorder.
Fabry disease, an X-linked condition affecting the metabolism of lipids, can lead to severe health complications, including heart problems, kidney failure, and stroke. The current treatment requires patients to receive 1 mg/kg of pegunigalsidase alfa bi-weekly. However, the newly validated regimen proposes administering 2 mg/kg every four weeks, enhancing convenience for patients.
The variation submission is backed by promising results from the completed Phase 3 study PB-102-F50, which showed that the new dosing schedule could maintain therapeutic benefits for adults who previously received more frequent doses. These results were recently published in the respected Journal of Inherited Metabolic Disease.
This achievement marks a pivotal moment in the ongoing effort to alleviate the burden of treatment for adults with Fabry disease. "This validation is a testament to our commitment to addressing the unmet medical needs of these patients," expressed Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases.
Meanwhile, Dror Bashan, Protalix's President and CEO, emphasized their dedication to exploring beneficial treatment alternatives. Their joint commitment to the rare disease community will pave the way for further therapeutic advancements aimed at enhancing patient wellbeing.
Given the complexities surrounding Fabry disease treatment, the updated guidelines also emphasize the importance of safety. Patients receiving pegunigalsidase alfa are cautioned about potential hypersensitivity reactions. Careful monitoring and immediate medical support are advised for those experiencing any severe reactions. Health practitioners are encouraged to prepare for emergencies related to anaphylaxis and other hypersensitivity events.
Chiesi's mission extends beyond just providing treatments; it's about ensuring accessibility for all patients affected by rare diseases. The company aims for equitable access, aspiring to improve the overall quality of life for individuals grappling with Fabry disease and their families.
As part of its commitment to sustainability, Chiesi has transitioned to a Benefit Corporation in several countries, including Italy and the US. This legal status enforces a high standard for societal and environmental responsibility. By prioritizing innovative solutions that consider patients' needs, Chiesi Group continues to be a noteworthy player in the biopharmaceutical space.
Protalix, utilizing its unique ProCellEx® plant-based expression platform, is also advancing therapeutic developments with a focus on sustainable practices. The company has received FDA and EMA approval for its two flagship products, underlining its footing in the biopharmaceutical landscape.
With this collaborative endeavor, Chiesi and Protalix demonstrate a profound dedication to shaping the future of rare disease treatments, promising new solutions for those affected by Fabry disease and inspiring hope in the healthcare community altogether.
Fabry disease, an X-linked condition affecting the metabolism of lipids, can lead to severe health complications, including heart problems, kidney failure, and stroke. The current treatment requires patients to receive 1 mg/kg of pegunigalsidase alfa bi-weekly. However, the newly validated regimen proposes administering 2 mg/kg every four weeks, enhancing convenience for patients.
The variation submission is backed by promising results from the completed Phase 3 study PB-102-F50, which showed that the new dosing schedule could maintain therapeutic benefits for adults who previously received more frequent doses. These results were recently published in the respected Journal of Inherited Metabolic Disease.
This achievement marks a pivotal moment in the ongoing effort to alleviate the burden of treatment for adults with Fabry disease. "This validation is a testament to our commitment to addressing the unmet medical needs of these patients," expressed Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases.
Meanwhile, Dror Bashan, Protalix's President and CEO, emphasized their dedication to exploring beneficial treatment alternatives. Their joint commitment to the rare disease community will pave the way for further therapeutic advancements aimed at enhancing patient wellbeing.
Given the complexities surrounding Fabry disease treatment, the updated guidelines also emphasize the importance of safety. Patients receiving pegunigalsidase alfa are cautioned about potential hypersensitivity reactions. Careful monitoring and immediate medical support are advised for those experiencing any severe reactions. Health practitioners are encouraged to prepare for emergencies related to anaphylaxis and other hypersensitivity events.
Chiesi's mission extends beyond just providing treatments; it's about ensuring accessibility for all patients affected by rare diseases. The company aims for equitable access, aspiring to improve the overall quality of life for individuals grappling with Fabry disease and their families.
As part of its commitment to sustainability, Chiesi has transitioned to a Benefit Corporation in several countries, including Italy and the US. This legal status enforces a high standard for societal and environmental responsibility. By prioritizing innovative solutions that consider patients' needs, Chiesi Group continues to be a noteworthy player in the biopharmaceutical space.
Protalix, utilizing its unique ProCellEx® plant-based expression platform, is also advancing therapeutic developments with a focus on sustainable practices. The company has received FDA and EMA approval for its two flagship products, underlining its footing in the biopharmaceutical landscape.
With this collaborative endeavor, Chiesi and Protalix demonstrate a profound dedication to shaping the future of rare disease treatments, promising new solutions for those affected by Fabry disease and inspiring hope in the healthcare community altogether.
Topics Health）
【About Using Articles】
You can freely use the title and article content by linking to the page where the article is posted.
※ Images cannot be used.
【About Links】
Links are free to use.