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Protalix and Chiesi Resubmit FDA Application For Rare Disease Treatment

PLX | November 30, 2022

(NewsDirect)

Protalix BioTherapeutics, Inc. (NYSE AMERICAN: PLX), an Israeli biopharmaceutical company, and Chiesi Global Rare Diseases, a business unit of the Chiesi Group, have resubmitted a Biologics License Application (BLA) for pegunigalsidase alfa (PRX–102), a drug candidate for the treatment of adult patients with Fabry disease.

Fabry disease is a rare enzyme deficiency that afflicts 1 in 40,000 to 60,000 people worldwide. Patients with Fabry are unable to break down the fatty substance globotriaosylceramide (Gb3), leading to accumulation of Gb3 in the blood vessels and symptoms including impaired blood circulation, end-organ failure, and increased risk of heart attack and stroke.

"The resubmission of the BLA represents a significant milestone for Protalix and we believe it has meaningful potential for patients and families affected by Fabry disease,” said Dror Bashan, Protalix's President and Chief Executive Officer. "Together with Chiesi, we are committed to continuing to work with the FDA toward our goal of achieving regulatory approval and making PRX–102 available to patients with this rare disease in the United States."

A Step Closer To Important Treatment

Protalix develops treatments from its proprietary plant cell-based expression system, ProCellEx®. Its drug candidate PRX-102 is a modified recombinant a–Galactosidase–A enzyme that would allow the Fabry disease patient to digest Gb3. This drug is part of Protalix’s novel PEGylated enzyme replacement therapy (ERT) treatment.

The resubmission of the BLA for drug candidate PRX-102 involves a rigorous and comprehensive set of data on the therapy. Protalix is confident moving forward, as it was the first company to receive Food and Drug Administration (FDA) approval of a protein produced through a plant cell-based expression system. Now it is collaborating with the rare disease unit of the Chiesi Group, an Italian-based therapeutic development company, to develop and commercialize PRX-102 for the treatment of Fabry disease.

“We believe it is important to deliver a potential new treatment option," said Giacomo Chiesi, head of Chiesi Global Rare Diseases, commenting on the resubmission of the BLA. "Together with Protalix, we thank the investigators and study participants who have made reaching this milestone possible and have supported our joint commitment to bringing this new treatment option to the Fabry patient community."

Protalix anticipates the FDA will review the resubmission within the next 6 months.

Other companies looking to develop treatments for Fabry disease include Avrobio Inc. (NASDAQ: AVRO), Freeline Therapeutics Holdings PLC (NASDAQ: FRLN), and Sangamo Therapeutics Inc. (NASDAQ: SGMO).

To learn more about Protalix BioTherapeutics, click here.

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. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified stabilized version of the recombinant human a–Galactosidase–A protein for the treatment of Fabry disease; alidornase alfa or PRX–110, for the treatment of various human respiratory diseases or conditions; PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of severe gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.

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