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Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce EMA Submission Validation for Pegunigalsidase Alfa

Chiesi Global Rare Diseases, a segment of the Chiesi Group dedicated to developing innovative treatments for individuals with rare conditions, recently announced a significant milestone. The European Medicines Agency (EMA) has validated their Variation Submission concerning Pegunigalsidase alfa. This submission supports a novel, less frequent dosing regimen of 2 mg/kg body weight every four weeks aimed specifically at adult patients with Fabry disease.

Traditionally, the approved dosage of Pegunigalsidase alfa has been 1 mg/kg, administered bi-weekly. This new proposal is especially focused on patients who have previously received treatment with agalsidase-alfa or agalsidase-beta. The changes are supported by a detailed Population-PK model, alongside exposure-response analyses derived from clinical trials.

Clinical Background and Importance

The clinical foundation of this new proposal comes from both the completed Phase 3 study, PB-102-F50 (BRIGHT), and its ongoing extension study, CLI-06657AA1-03. Results from the PB-102-F50 trial have recently been published in the Journal of Inherited Metabolic Disease, underscoring the progress in understanding the efficacy of Pegunigalsidase alfa. The potential to offer a dosing regimen that allows for less frequent treatment is crucial for many adult patients who often grapple with the burden of regular treatment schedules.

Giancomo Chiesi, Executive Vice President at Chiesi Global Rare Diseases, celebrated this validation as a pivotal moment, emphasizing their commitment to addressing the unmet needs of those living with Fabry disease. He stated, “This application’s validation represents a vital step forward for adult patients suffering from Fabry disease who often face pressing healthcare needs.”

Dror Bashan, President and CEO of Protalix BioTherapeutics, expressed his optimism regarding the revised dosage, indicating that the new regimen may offer a beneficial alternative for those affected by the disease. Bashan reaffirmed their joint commitment with Chiesi to continue advocating for patients and developing innovative therapeutic options.

Collaborative Commitment to Rare Diseases

Chiesi Global Rare Diseases is not just about creating treatments; the group aims to generate a world where effective therapies are accessible for all rare diseases. With a foundational belief in health equity, their mission is deeply rooted in ensuring accessible healthcare solutions to the rare disease community.

Chiesi operates as a Benefit Corporation, striving to generate social and environmental impact alongside their business pursuits. Their commitment is further evidenced by achieving B Corp certification, indicating adherence to high standards for societal contributions.

Protalix BioTherapeutics operates under a similar ethos, focusing on innovative biopharmaceutical developments, utilizing their proprietary ProCellEx® plant cell-based expression system for therapeutic products. Pegunigalsidase alfa is significant as it's recognized as the first successfully approved protein through a plant-based system—marking a new dimension in biopharmaceutical manufacturing.

In conjunction, both companies are excited about the prospect of continuing collaborations with regulatory bodies to ensure that innovative solutions meet the evolving needs of the rare diseases community, particularly in the context of ongoing healthcare challenges worldwide.

The collaborative efforts of Chiesi and Protalix exemplify a forward-thinking approach to healthcare solutions, promising improved patient experiences through reduced treatment frequencies and increased access to necessary therapies.

As the agencies continue to evaluate and approve innovative therapies, the future for adult patients with Fabry disease looks promising, with Chiesi and Protalix aiming to be at the forefront of change in therapeutic practices.