Breaking News: European Medicines Agency Validates Variation Submission for PEGUNIGALSIDASE ALFA
Application to label a less frequent dosing regimen at a dose of 2 mg/kg body weight administered every four weeks in adult patients with Fabry disease in European Union
PARMA, Italy and CARMIEL Israel, Dec. 9, 2024
Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, and Protalix BioTherapeutics, Inc., a biopharmaceutical company focused on the development, production, and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today announced that the European Medicines Agency (EMA) has validated the Variation Submission for pegunigalsidase alfa to label a less frequent dosing regimen at a dose of 2 mg/kg body weight administered every four weeks in adult patients with Fabry disease. The currently approved dose of pegunigalsidase alfa is 1 mg/kg administered every two weeks.
This validation of the Variation Submission is a significant milestone in the treatment of Fabry disease, a rare genetic disorder that results from the buildup of a particular type of fat in the body’s cells. Patients with Fabry disease often experience a range of symptoms, including pain, kidney problems, and heart issues. Pegunigalsidase alfa has been shown to be effective in reducing the accumulation of this fat in patients with Fabry disease.
The approval of a less frequent dosing regimen for pegunigalsidase alfa provides adult patients with Fabry disease in the European Union with a more convenient treatment option. By reducing the frequency of administration to once every four weeks, patients may experience improved adherence to their treatment plan and potentially better outcomes in managing their condition.
Chiesi Global Rare Diseases and Protalix BioTherapeutics are committed to advancing the treatment options available to patients with rare diseases, and the validation of the Variation Submission for pegunigalsidase alfa represents a step forward in meeting the needs of this patient population.
Effect on Me:
As a patient with Fabry disease, the validation of the Variation Submission for pegunigalsidase alfa to label a less frequent dosing regimen at a higher dose provides me with a more convenient and potentially more effective treatment option. The ability to receive treatment once every four weeks, as opposed to every two weeks, may improve my adherence to the prescribed regimen and lead to better management of my condition.
Effect on the World:
The validation of the Variation Submission for pegunigalsidase alfa by the European Medicines Agency represents a significant advancement in the treatment of Fabry disease on a global scale. By offering a less frequent dosing regimen at a higher dose for adult patients with Fabry disease in the European Union, this update has the potential to improve the quality of life for individuals living with this rare genetic disorder. It also highlights the importance of continued research and development efforts in the field of rare diseases.
Conclusion
In conclusion, the validation of the Variation Submission for pegunigalsidase alfa by the European Medicines Agency is a positive development for patients with Fabry disease in the European Union. This update provides a more convenient treatment option with a less frequent dosing regimen at a higher dose, potentially leading to improved adherence and better outcomes for individuals living with this rare genetic disorder. Chiesi Global Rare Diseases and Protalix BioTherapeutics continue to demonstrate their commitment to advancing innovative therapies for rare diseases, and this milestone marks a significant step forward in meeting the needs of patients with Fabry disease.
