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CARMIEL, Israel and BOSTON, Feb. 24, 2022 /PRNewswire/ — Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: 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, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici SpA, an international research-focused healthcare Group (Chiesi Group), today announced the submission of a Marketing Authorization Application (MAA) via centralized procedure to the European Medicines Agency (EMA) for pegunigalsidase alfa (PRX–102) for the proposed treatment of adults with Fabry disease, and the subsequent validation of the MAA by the EMA.
The MAA submission includes a comprehensive set of preclinical, clinical and manufacturing data compiled from the Company’s completed and ongoing clinical studies evaluating PRX–102 as a potential treatment for Fabry disease. The submission is supported by the 12–month interim data analysis generated from the phase III BALANCE clinical trial, which was released in June 2021. Data generated from the completed phase III BRIDGE clinical trial, the phase 1/2 clinical trial in naive or untreated patients, and from the related extension studies with 1 mg/kg every two weeks were also included in the filing. In addition, the MAA includes data from the completed 12–month switch–over phase III BRIGHT clinical trial of patients treated with the 2 mg/kg every 4 weeks dosage.
“The submission marks a considerable accomplishment in the development of PRX–102 and is an important step forward in navigating regulatory channels in the European Union. This centralized procedure via the EMA allows for the submission of a single marketing authorization application to the European Union, and, if approved, allows Chiesi, our commercial partner, to market and make PRX–102 available to patients and healthcare professionals across the entire European Union,” said Dror BashanProtalix’s President and Chief Executive Officer. “We are committed to bringing PRX–102 to market and look forward to providing an alternative treatment option for people with Fabry disease.”
The scientific evaluation will be conducted by the Committee for Medicinal Products for Human Use (CHMP) with predefined assessment milestones. At the completion of the review, the CHMP will issue a scientific opinion on whether PRX-102 may be authorized or not. The EMA will forward this opinion to the European Commission, which is expected to adopt the EMA’s scientific opinion.
“Our team at Chiesi is deeply committed to the Fabry disease community and we are grateful to the patients and investigators who have helped us better understand the unmet treatment needs and whose participation in the clinical trials has led us to this important milestone,” said Giacomo ChiesiHead of Chiesi Global Rare Diseases. “We believe that the safety and efficacy data demonstrated by PRX-102 in clinical trials strongly supports this application and we look forward to completing the final stages of regulatory review.”
About Fabry Disease
Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person’s body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood and in the blood vessel walls. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end–organ failure …
Full story available on Benzinga.com
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