Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce Validation of Variation Submission by European Medicines Agency for pegunigalsidase alfa
Chiesi Global Rare Diseases and Protalix BioTherapeutics (PLX) announced the European Medicines Agency's validation of their Variation Submission for pegunigalsidase alfa. The submission aims to approve a less frequent dosing regimen of 2 mg/kg every four weeks for adult Fabry disease patients, compared to the current 1 mg/kg every two weeks.
The application is supported by a revised Population-PK model, exposure-response analyses, and clinical data from the Phase 3 BRIGHT study and its ongoing extension study. The studies investigated the 2 mg/kg every four weeks dosing in adult Fabry disease patients previously treated with agalsidase-alfa or -beta.
Chiesi Global Rare Diseases e Protalix BioTherapeutics (PLX) hanno annunciato la validazione da parte dell'Agenzia Europea dei Medicinali della loro Richiesta di Variazione per pegunigalsidase alfa. La richiesta mira ad approvare un regime di dosaggio meno frequente di 2 mg/kg ogni quattro settimane per i pazienti adulti affetti da malattia di Fabry, rispetto all'attuale 1 mg/kg ogni due settimane.
La domanda è supportata da un modello Population-PK rivisitato, analisi esposizione-risposta e dati clinici dallo studio di Fase 3 BRIGHT e dal suo studio di estensione in corso. Gli studi hanno esaminato il dosaggio di 2 mg/kg ogni quattro settimane in pazienti adulti con malattia di Fabry precedentemente trattati con agalsidase-alfa o -beta.
Chiesi Global Rare Diseases y Protalix BioTherapeutics (PLX) anunciaron la validación por parte de la Agencia Europea de Medicamentos de su Solicitud de Variación para pegunigalsidase alfa. La solicitud tiene como objetivo aprobar un régimen de dosificación menos frecuente de 2 mg/kg cada cuatro semanas para pacientes adultos con enfermedad de Fabry, en comparación con el actual 1 mg/kg cada dos semanas.
La aplicación cuenta con el apoyo de un modelo Population-PK revisado, análisis de exposición-respuesta y datos clínicos del estudio de Fase 3 BRIGHT y su estudio de extensión en curso. Los estudios investigaron la dosificación de 2 mg/kg cada cuatro semanas en pacientes adultos con enfermedad de Fabry previamente tratados con agalsidase-alfa o -beta.
Chiesi Global Rare Diseases와 Protalix BioTherapeutics (PLX)는 유럽 의약품청이 pegunigalsidase alfa에 대한 변동 제출안을 검증했다고 발표했습니다. 이 제출안은 성인 Fabry 병 환자에게 현재의 1 mg/kg 2주마다 투여하는 것에 비해 4주마다 2 mg/kg의 덜 빈번한 투여 요법을 승인하는 것을 목표로 합니다.
이 신청서는 개정된 Population-PK 모델, 노출-반응 분석 및 3상 BRIGHT 연구와 계속 진행 중인 연장 연구의 임상 데이터를 지원합니다. 해당 연구들은 agalsidase-alfa 또는 -beta로 이전에 치료받은 성인 Fabry 병 환자에게 4주마다 2 mg/kg 투여의 효과를 조사했습니다.
Chiesi Global Rare Diseases et Protalix BioTherapeutics (PLX) ont annoncé la validation par l'Agence Européenne des Médicaments de leur demande de variation pour le pegunigalsidase alfa. Cette demande vise à approuver un schéma posologique moins fréquent de 2 mg/kg toutes les quatre semaines pour les patients adultes atteints de la maladie de Fabry, par rapport au régime actuel de 1 mg/kg toutes les deux semaines.
La demande est soutenue par un modèle Population-PK révisé, des analyses exposition-réponse et des données cliniques de l'étude de Phase 3 BRIGHT et de son étude d'extension en cours. Les études ont examiné la posologie de 2 mg/kg toutes les quatre semaines chez les patients adultes atteints de la maladie de Fabry précédemment traités par agalsidase-alfa ou -beta.
Chiesi Global Rare Diseases und Protalix BioTherapeutics (PLX) haben die Validierung ihrer Änderungsanmeldung für pegunigalsidase alfa durch die Europäische Arzneimittelagentur bekannt gegeben. Die Anmeldung zielt darauf ab, ein weniger häufiges Dosierungsschema von 2 mg/kg alle vier Wochen für erwachsene Patienten mit Fabry-Krankheit zu genehmigen, im Vergleich zu den derzeitigen 1 mg/kg alle zwei Wochen.
Der Antrag wird durch ein überarbeitetes Population-PK-Modell, Expositions-Wirkungs-Analysen und klinische Daten aus der Phase-3-Studie BRIGHT sowie deren laufende Verlängerungsstudie unterstützt. Die Studien untersuchten die Dosierung von 2 mg/kg alle vier Wochen bei erwachsenen Fabry-Krankheitspatienten, die zuvor mit Agalsidase-Alfa oder -Beta behandelt wurden.
- EMA validation of variation submission represents regulatory progress
- Potential for less frequent dosing regimen could improve patient convenience
- Application supported by completed Phase 3 study data
- None.
Insights
The EMA validation of Protalix's variation submission for pegunigalsidase alfa represents a significant development in Fabry disease treatment. The proposed change from bi-weekly to monthly dosing at 2mg/kg could substantially improve patient compliance and quality of life. This modification is backed by robust clinical data from the Phase 3 BRIGHT study and its extension, demonstrating the scientific validity of the less frequent dosing regimen.
The potential approval would give Protalix and Chiesi a competitive advantage in the Fabry disease market, as reduced dosing frequency is a key differentiator in enzyme replacement therapies. This could lead to increased market share and improved commercial performance, particularly in the European market where patient convenience is highly valued.
From a market perspective, this regulatory milestone could significantly enhance the commercial potential of pegunigalsidase alfa in Europe. The less frequent dosing schedule addresses a critical market need for reduced treatment burden, potentially leading to better market penetration and patient adoption. For PLX shareholders, this development could positively impact the company's market position in the
The collaboration with Chiesi, a well-established rare disease player, provides strong commercialization support. If approved, the new dosing regimen could strengthen the product's competitive position against existing treatments from Sanofi and Takeda, potentially driving revenue growth for both companies.
- 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 /PRNewswire/ -- 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. (NYSE American: PLX), 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.
The variation application is supported by a revised Population-PK model and new exposure-response analyses and by the clinical data on pegunigalsidase alfa 2 mg/kg E4W from the completed Phase 3 study PB-102-F50 (BRIGHT) and its ongoing extension study CLI-06657AA1-03 (formerly known as PB-102-F51), that have investigated the 2 mg/kg every four weeks dosing regimen in adult patients with Fabry disease who were previously treated with agalsidase-alfa or -beta administered every two weeks. Results of the completed study PB-102-F50 were recently published in the Journal of Inherited Metabolic Disease.
"The validation of this variation application is an important milestone in our efforts to reduce the burden of treatment for some adult patients living with Fabry disease who continue to experience unmet medical needs," said Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases. "We are committed to delivering innovative therapies and solutions for people living with Fabry disease, their families and caregivers."
"Based on study results, we believe in the potential of pegunigalsidase alfa 2 mg/kg administered every four weeks to be a beneficial, alternative dosing option for some adults living with Fabry disease," said Dror Bashan, Protalix's President and Chief Executive Officer. "Together with Chiesi, we remain committed to meeting the needs of people with Fabry disease and bringing additional therapeutic options to market. We look forward to continuing to work closely with the agency in the months ahead."
Indication and Important Safety Information for Elfabrio® (pegunigalsidase alfa-iwxj)
Indication
Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease.
Important Safety Information
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS |
Patients treated with Elfabrio have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Elfabrio administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Elfabrio may be considered. |
Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs) and instruct them to seek medical care immediately if such symptoms occur.
- If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment.
- If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose.
In clinical trials, 20 (
In clinical trials, 41 (
A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted.
When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels).
The most common adverse reactions (≥
Please see Full Prescribing Information for Elfabrio.
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
About Chiesi Group
Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment.
By changing its legal status to a Benefit Corporation in
With over 85 years of experience, Chiesi is headquartered in
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. It is the first company to gain FDA approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix's second product, pegunigalsidase alfa, was approved by both the FDA and the EMA in May 2023.
Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of pegunigalsidase alfa.
Protalix BioTherapeutics' Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on Protalix's current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: risks related to the commercialization of pegunigalsidase alfa (pegunigalsidase alfa-iwxj), Protalix's approved product for the treatment of adult patients with Fabry disease; risks relating to pegunigalsidase alfa market acceptance, competition, reimbursement and regulatory actions, including as a result of the boxed warning contained in the FDA approval received for the product; the possible disruption of Protalix's operations due to the war declared by
Chiesi Group Media Contacts
Chiara Travagin
Head of Communications, Rare Diseases
Tel: +39 348 8818985
Email: c.travagin@chiesi.com
Adam Daley
Berry & Company Public Relations
Tel: +1 212 253 8881
Email: adaley@berrypr.com
Protalix Investor Contact
Mike Moyer, Managing Director
LifeSci Advisors
Tel: +1-617-308-4306
Email: mmoyer@lifesciadvisors.com
PP-RA-00423 V1.0
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SOURCE Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc.
FAQ
What is the new dosing regimen proposed for pegunigalsidase alfa (PLX) in the EMA submission?
What clinical studies support Protalix's (PLX) EMA variation submission for pegunigalsidase alfa?
What is the current approved dosing schedule for Protalix's (PLX) pegunigalsidase alfa?
