Benzinga | Oct 11, 2021 06:51AM EDT

Protalix Biotherapeutics And Chiesi Global Rare Diseases Provide Regulatory Update On PRX-102 For The Treatment Of Fabry Disease

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(r) plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), today provided a regulatory update regarding pegunigalsidase alfa (PRX--102) for the proposed treatment of Fabry disease which included an announcement of the receipt of the official Type A (End-of-Review) meeting minutes from the U.S. Food and Drug Administration (FDA) regarding the Complete Response Letter (CRL) received for the PRX--102 Biologics License Application (BLA) confirming a potential pathway for resubmission of a BLA for PRX--102.

"We are pleased with the results of the Type A meeting," said Dror Bashan, Protalix's President and Chief Executive Officer. "We remain committed to the Fabry community and our goal of providing an alternative treatment option for Fabry patients."

The Type A meeting was held on September 9, 2021. As part of the meeting minutes provided by the FDA, which included the preliminary comments and meeting discussion, the FDA, in principle, agreed that the data package proposed to the FDA for the BLA resubmission has the potential to support a traditional approval of PRX--102 for the treatment of Fabry disease. The planned data package for the BLA resubmission, given the changed regulatory landscape in the United States, will include the final two--year analyses of the phase III BALANCE clinical trial.

"We are encouraged by the productive discussion with the FDA, which we believe provides a pathway to resubmit the PRX--102 BLA, and appreciate the valuable feedback and guidance provided," said Einat Brill Almon, Ph.D., Protalix's Sr. Vice President and Chief Development Officer. "We look forward to our continued development of PRX--102 and our collaborative efforts with Chiesi in advancing the PRX--102 program towards commercialization."

Protalix and Chiesi also announced that a meeting was held with the Rapporteur and Co-Rapporteur of the European Medicines Agency (EMA) on October 8, 2021 regarding PRX--102. At the meeting, Chiesi and Protalix discussed the scope of the anticipated Marketing Authorization Application (MAA) for the European Union, and the Rapporteur and Co-Rapporteur were generally supportive of a planned MAA submission for PRX--102. This is an important step in the necessary pre-submission activities leading up to a planned MAA submission. Chiesi and Protalix expect to submit an MAA for PRX--102 during the first quarter of 2022.

"On behalf of our team at Chiesi, we thank the patients, families and clinicians for their participation in our clinical studies evaluating PRX--102. We remain committed to advancing this important program to expand support for the Fabry disease community," said Giacomo Chiesi, Head of Chiesi Global Rare Diseases.

About Fabry Disease

Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal ?--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 ?--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 -- particularly of the kidneys, but also of the heart and the cerebrovascular system.

About Pegunigalsidase Alfa (PRX--102)

Pegunigalsidase alfa (PRX--102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant ?--Galactosidase--A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX--102 has been observed to have a circulatory half-life of approximately 80 hours. Protalix designed PRX--102 to potentially address the continued unmet clinical need in Fabry patients.

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. 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 ?--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 refractory 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.