Protalix BioTherapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the development, production and commercialization of recombinant therapeutic proteins produced via its proprietary ProCellEx plant cell-based protein expression system.
The company is the first and only organization to gain U.S. Food and Drug Administration, or FDA, approval of a protein produced through plant cell-based expression in suspension. The company’s unique expression system represents a new metho...
Protalix BioTherapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the development, production and commercialization of recombinant therapeutic proteins produced via its proprietary ProCellEx plant cell-based protein expression system.
The company is the first and only organization to gain U.S. Food and Drug Administration, or FDA, approval of a protein produced through plant cell-based expression in suspension. The company’s unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
As of December 31, 2024, the company had successfully developed two commercial products, both of which are enzyme replacement therapies (ERTs): Elfabrio (pegunigalsidase alfa) for the treatment of adult patients with a confirmed diagnosis of Fabry disease, and Elelyso (taliglucerase alfa) for the treatment of adult patients with Gaucher disease. Elfabrio, which the company referred to as PRX-102 during its development stage, has been approved for marketing in the United States, the European Union, Great Britain, Switzerland, Peru, Israel, Russia, and Singapore. The company has partnered with Chiesi for the development and commercialization of Elfabrio.
The company has licensed the rights to commercialize Elelyso worldwide (other than Brazil) to Pfizer, and in Brazil to Fiocruz. Elelyso is marketed as BioManguinhos alfataliglicerase in Brazil.
The company is committed to leveraging its track record of success as it progresses with the development of treatments for rare and orphan diseases. In addition, the company continuously works on the further development and enhancement of its ProCellEx technology. Accordingly, the company is turning its focus to new, early-stage product candidates that treat indications for which there are high unmet needs in terms of efficacy and safety, including renal diseases. The company intends to use its ProCellEx platform and PEGylation capabilities, as well as other modalities, such as small molecules and antibodies, to take advantage of highly innovative opportunities. The company is also exploring novel platform technologies.
Elfabrio was the subject of a phase III clinical program. The phase III clinical program included three separate studies, which are referred to as the BALANCE study, the BRIDGE study, and the BRIGHT study. The phase III clinical program analyzed two potential dosing regimens: 1 mg/kg every two weeks and 2 mg/kg every four weeks. In addition, the phase III clinical program included two extension studies in which subjects that participated in its phase I/II clinical trials and phase III clinical trials had the opportunity to enroll and continue to be treated with PRX-102.
The Biologics License Application, or BLA, for Elfabrio for the treatment of adult patients with Fabry disease was resubmitted to the FDA on November 9, 2022. An initial BLA for Elfabrio was submitted to the FDA on May 27, 2020, under the FDA’s Accelerated Approval pathway, but resulted in a Complete Response Letter, or CRL. A Marketing Authorization Application, or MAA, was submitted to the EMA on February 7, 2022, after the October 8, 2021, meeting the company held, together with Chiesi, with the Rapporteur and Co-Rapporteur of the EMA regarding PRX-102.
On May 5, 2023, the European Commission, or EC, announced that it had approved the MAA for Elfabrio, and on May 9, 2023, the FDA announced that it had approved the Biologics License Application, or BLA, for Elfabrio (pegunigalsidase alfa-iwxj) injection BLA 761161, each for adult patients with a confirmed diagnosis of Fabry disease. Both approvals cover the 1 mg/kg every two weeks dosage. Elfabrio was approved by the FDA with a boxed warning for hypersensitivity reactions/anaphylaxis, consistent with ERT class labeling, and Warnings/Precautions providing guidance on the signs and symptoms of hypersensitivity and infusion-associated reactions seen in the clinical studies, as well as treatments to manage such events should they occur.
The FDA publicly released the internal review documents for Elfabrio. These documents provide previously unavailable additional information regarding the basis for the FDA’s May 2023 approval decision. In particular, the FDA determined that substantial evidence of effectiveness for Elfabrio in Fabry patients was established with one adequate and well-controlled study (the company’s phase I/II clinical trial of Fabry Disease, Study PB-102-F01/02) with confirmatory evidence provided by the company’s phase III BALANCE clinical trial of PRX-102 for the treatment of Fabry disease, or the BALANCE study (also referred to as Study PB-102-F20).
The company’s first product, Elelyso, an ERT for the treatment of patients with Gaucher disease, was first approved by the FDA in May 2012 and is now approved for marketing in 23 markets, including Brazil, Israel, and others.
In addition, the company is developing PEGylated uricase, or PRX-115, for the treatment of uncontrolled gout; Long Acting (LA) DNase I, or PRX-119, for the treatment of NETs-related diseases; and a number of other technologies and preclinical assets. The company has completed a phase I First-in-Human clinical trial of PRX-115, and it expects to commence a phase II clinical trial of PRX-115 in the second half of 2025.
The company’s proprietary ProCellEx platform is being used to manufacture both of its approved and marketed products, as well as PRX-115 and PRX-119.
The company’s strategy moving forward is to develop proprietary recombinant proteins designed to address high unmet needs in the genetic and non-genetic rare disease space that are therapeutically superior to existing recombinant proteins currently marketed for the same indications. Consistent with this strategy, the company has a number of product candidates in varying stages of the clinical development process.
Product Pipeline
ProCellEx: The company’s Proprietary Protein Expression System
ProCellEx is the company’s proprietary platform used to produce and manufacture recombinant proteins through plant cell-based expression in suspension. The company is the first and only organization to receive FDA approval of a protein produced through plant cell-based expression, and with the approval of Elfabrio, the company now produces two commercial proteins through its platform.
ProCellEx consists of a comprehensive set of proprietary technologies and capabilities, including the use of advanced genetic engineering and plant cell culture technology, enabling the company to produce complex, proprietary, and biologically equivalent proteins for a variety of human diseases. The company’s protein expression system facilitates the creation and selection of high-expressing, genetically stable cell lines capable of expressing recombinant proteins. The system plays an important role in the execution of the company’s corporate strategy, as it allows the company to develop and produce tailored complex recombinant therapeutic proteins and to genetically engineer and/or chemically modify such proteins pre- and/or post-production.
The company’s ProCellEx technology allows for many unique advantages, including: biologic optimization; an ability to handle complex protein expressions; flexible manufacturing with improvements through efficiencies, enhancements, and/or rapid horizontal scale-ups; a simplified production process; elimination of the risk of viral contaminations from mammalian components; and intellectual property advantages.
The company developed ProCellEx based on its plant cell culture technology for the development, expression, and manufacture of recombinant proteins that are the essential foundation of modern biotechnology. The company develops new, recombinant therapeutic proteins by using the natural capability of agrobacterium to transfer a DNA fragment into the plant chromosome, allowing the genome of the plant cell to code for specific proteins of interest.
The company’s ProCellEx technology can be utilized to express complex therapeutic proteins belonging to different drug classes, such as enzymes, hormones, monoclonal antibodies, cytokines, and vaccines. The company’s plant cell culture technology uses cells, such as carrot and tobacco (BY-2) cells, which undergo advanced genetic engineering and/or chemical modifications, and are grown on an industrial scale in a disposable, flexible bioreactor system.
The ProCellEx reactors are easy to use and maintain, allow for rapid horizontal scale-up, and do not involve the risk of mammalian viral contamination. The company’s bioreactors are well-suited for plant cell growth using a simple, inexpensive, chemically defined growth medium.
In addition, the company continuously works on the further development and enhancement of its ProCellEx technology.
ProCellEx: Protalix’s Differentiated Plant Cell Protein Expression Platform
Business Highlights
The company has two commercial products, each of which is an ERT: Elelyso and Elfabrio. The company’s pipeline of product candidates includes PEGylated uricase for the treatment of uncontrolled gout, Long Acting (LA) DNase I for the treatment of NETs, and other technologies and preclinical assets.
Elelyso (taliglucerase alfa) for the Treatment of Gaucher Disease
Elelyso for the treatment of Gaucher disease is currently approved and marketed in 23 countries, including the United States, Australia, Canada, Israel, Brazil, Russia, and Turkey. In June 2012, the EMA’s Committee for Medicinal Products for Human Use, or the CHMP, issued a positive opinion regarding the benefit of Elelyso but did not immediately grant marketing authorization because of the ten-year market exclusivity granted to Vpriv (Takeda Shire) in August 2010 for the same condition, which was extended for an additional two years, and expired in August 2022. The company has granted the marketing rights to Elelyso globally, excluding Brazil, to Pfizer through an exclusive licensing agreement. The company maintains the distribution rights to Elelyso in Brazil, where it is currently marketed as BioManguinhos alfataliglicerase, through the Supply and Technology Transfer Agreement, or the Brazil Agreement, it entered into on June 18, 2013, with Fiocruz, an arm of the Brazilian MoH.
Elfabrio (pegunigalsidase alfa or PRX-102) for the Treatment of Fabry Disease
Elfabrio for the treatment of adult patients with Fabry disease is the company’s proprietary, plant cell culture expressed enzyme, and a chemically modified stabilized version of the recombinant alpha-Galactosidase-A protein, a lysosomal enzyme. Elfabrio has been approved by the FDA for marketing in the United States, the European Union, Great Britain, Switzerland, Peru, Israel, Russia, and Singapore.
Protalix Ltd., the company’s wholly owned subsidiary, has entered into two exclusive licensing and supply agreements with Chiesi for Elfabrio, one global excluding the United States, and the second for the United States.
PEGylated Uricase (PRX-115)
PRX-115 is the company’s plant cell-expressed recombinant PEGylated uricase (urate oxidase) – a chemically modified enzyme under development for the potential treatment of uncontrolled gout. Based on market research the company has commissioned, it estimates that approximately 25% of the gout population in the U.S. and Western Europe does not have their gout controlled. Some of those patients cannot be treated with existing therapies; others stop treatment with existing therapies due to adverse events. In addition, such research presents that there are gout patients treated with existing therapies that continue to suffer from tophi despite having reached urate target levels.
PRX-119
PRX-119 is the company’s plant cell-expressed PEGylated recombinant human DNase I product candidate, which the company is designing to have an elongated half-life in the circulation for the potential treatment of NETs-related diseases. NETs, or Neutrophil extracellular traps, are web-like structures released by activated neutrophils that trap and kill a variety of microorganisms. NETs are composed of DNA, histones, antimicrobial, and pro-inflammatory proteins. The company’s proprietary modified DNase I, which the company has designed for long and customized systemic circulation in the bloodstream, may potentially enable effective treatment for these conditions.
In May 2024, the company announced its decision to expand its phase I First-in-Human clinical trial of PRX-115 by adding an eighth cohort with eight new patients and to commence preparations for a phase II clinical trial of PRX-115.
Marketed Products
Elelyso for the Treatment of Gaucher Disease
Elelyso (taliglucerase alfa), the company’s first commercial product, was approved by the FDA in 2012 for injection as an ERT for the long-term treatment of adult patients with a confirmed diagnosis of type 1 Gaucher disease. In August 2014, the FDA approved Elelyso for injection for pediatric patients. Elelyso is the first plant cell-derived recombinant protein to be approved by the FDA for the treatment of Gaucher disease and is now approved in 23 markets, including the United States, Brazil, Israel, and others.
Elfabrio for the Treatment of Fabry Disease
Elfabrio, the company’s second commercial product, was approved by the EC for marketing in the EU and by the FDA for marketing in the United States in May 2023 for adult patients with Fabry disease. Both approvals cover the 1 mg/kg every two weeks dosage. According to the EMA, overall, the benefit/risk balance of Elfabrio is positive in the claimed indication (Fabry disease). Similarly, the FDA review team concluded that in the context of Fabry disease as a rare, serious disease with limited therapeutic options that may not be suitable to all individual patients, the benefit-risk of Elfabrio is favorable for the treatment of adults with confirmed Fabry disease. The FDA noted its determination that substantial evidence of effectiveness for Elfabrio in Fabry patients was established with one adequate and well-controlled study, the company’s phase I/II clinical trial of Fabry disease, with confirmatory evidence provided by the BALANCE study. The FDA review team also concluded that the BALANCE study met its primary efficacy endpoint, which assessed the annualized rate of change in eGFR (estimated glomerular filtration rate) over 104 weeks.
Since the approvals by the FDA and the European Medicines Agency, or EMA, Elfabrio has been approved for marketing in Great Britain, Switzerland, Peru, Israel, Russia, and Singapore for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease.
Key Trials and Design
The company’s PRX-102 clinical development program was designed to show that PRX-102 has a potential clinical benefit in all adult Fabry patient populations when compared to the then-marketed Fabry disease enzymes, agalsidase beta and agalsidase alfa. In preclinical studies, PRX-102 showed significantly longer half-life due to higher enzyme stability, enhanced activity in Fabry disease-affected organs leading to a reduction of the accumulated substrate, and reduced immunogenicity, which together can potentially lead to improved efficacy through increased substrate clearance and significantly lower formation of anti-drug antibodies, or ADAs.
The phase III clinical program included three individual studies: the BALANCE study, the BRIDGE study, and the BRIGHT study. In the phase III clinical program overall, two potential dosing regimens for PRX-102 were analyzed: 1 mg/kg every two weeks, with the potential for improved efficacy and safety offering a potential alternative to existing enzyme replacement therapies, and 2 mg/kg every four weeks. The phase III program was preceded by the phase I/II clinical trial, a dose range-finding study in ERT-naïve adult patients with Fabry disease.
Phase III BALANCE Study
The BALANCE study (PB-102-F20, NCT02795676) was a pivotal 24-month, randomized, double-blind, active control study of PRX-102 in adult Fabry patients with deteriorating renal function designed to evaluate the safety and efficacy of 1 mg/kg of PRX-102 administered every two weeks compared to agalsidase beta. The Clinical Study Report for the BALANCE study, completed in July 2022, demonstrated a favorable tolerability profile. A total of 78 patients who were previously treated with agalsidase beta for at least one year, with an eGFR slope at screening worse than -2 mL/min/1.73 m2/year, were enrolled in the study. Patients were randomized on a 2:1 ratio for switching to PRX-102 or continuing on agalsidase beta. A total of 77 patients were treated; 52 with PRX-102 and 25 with agalsidase beta. Approximately 40% of the enrolled patients were female.
Forty-seven (90.4%) patients in the PRX-102 arm experienced at least one treatment-emergent adverse event (TEAE) compared to 24 (96.0%) in the agalsidase beta arm. The number of events adjusted to 100 years of exposure is 572.36 events for the PRX-102 arm and 816.85 events for the agalsidase beta arm.
TEAEs were reported for 21 (40.4%) patients in the PRX-102 arm compared to 11 (44.0%) in the agalsidase beta arm. The number of treatment-related events adjusted to 100 years of exposure is 42.85 events for the PRX-102 arm and 152.91 events for the agalsidase beta arm.
Considering that in the trial patients in the PRX-102 arm were exposed for the first time to the novel enzyme, tolerability data appear favorable for PRX-102 and in line with what was observed in the previous clinical studies of PRX-102.
The results of the direct, blinded comparison of PRX-102 to agalsidase beta, for the primary efficacy renal endpoints (i.e., eGFR change, eGFR slope) and for the main secondary endpoints (e.g., urine protein to creatinine ratio [UPCR], LVMI, MSSI, BPI) strongly suggest comparability in treatment effects between the two treatments.
At the same time, a potentially favorable safety profile was identified based on lower rates of IRR, lower ADA positivity, and less premedication use in the PRX-102 arm compared to agalsidase beta.
Phase III BRIDGE Study
The BRIDGE study (PB-102-F30, NCT03018730) was a 12-month open-label, single-arm switch-over study evaluating the safety and efficacy of PRX-102, 1 mg/kg infused every two weeks, in up to 22 Fabry patients previously treated with agalsidase alfa for at least two years and on a stable dose for at least six months. In the study, patients were screened and evaluated over three months while continuing agalsidase alfa treatment.
PRX-102 was well-tolerated in the BRIDGE study, with all adverse events being transient in nature without sequelae. The majority of TEAEs were mild or moderate in severity, with two patients (9.1%) withdrawing from the therapy due to hypersensitivity reactions that were resolved. The most common moderate TEAEs were nasopharyngitis, headache, and dyspnea. An immunogenicity assessment indicated that four out of 20 patients (20%) developed persistent ADAs over the course of the study, of which two had neutralizing activity.
Phase III BRIGHT Study
The BRIGHT study (PB-102-F50, NCT03180840) was a multicenter, multinational open-label, switch-over study designed to evaluate the safety, efficacy, and pharmacokinetics of treatment with 2 mg/kg of PRX-102 administered every four weeks for 52 weeks (a total of 14 infusions). The 2 mg/kg every four weeks dosage has not been approved by the EMA, FDA, or any other jurisdiction.
All 30 patients received at least one dose of PRX-102, and 29 patients completed the one-year study. Of these 29 patients, 28 received the intended regimen of 2 mg/kg every four weeks throughout the entire study, while one patient was switched to 1 mg/kg PRX-102 every two weeks per protocol at the 11th infusion.
Phase I/II Study
The phase I/II clinical trial of PRX-102 (NCT01678898) was a worldwide, multi-center, open-label, dose-ranging study designed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, and efficacy parameters of PRX-102 in adult patients with Fabry disease. It was completed in 2015.
Sixteen adult, naïve Fabry patients (9 male and 7 female) completed the phase I/II study, each in one of three dosing groups: 0.2 mg/kg, 1 mg/kg, and 2 mg/kg. Each patient received IV infusions of PRX-102 every two weeks for 12 weeks, with efficacy follow-up after six-month and twelve-month periods.
Data was recorded at 24 months from 11 patients who completed 12 months of the long-term open-label extension trial that succeeded the phase I/II study.
Extension Studies
Two long-term open-label extension studies were available for patients who completed the BALANCE, BRIDGE, and BRIGHT studies, and the extension of the phase I/II study. Overall, 126 patients who participated in the company’s PRX-102 clinical program initially opted, with the advice of the treating physician, to enroll in one of the extension studies: 97 patients in the 1 mg/kg every two weeks extension study (PB-102-F60, NCT03566017) (10 patients who completed an extension study from the phase I/II study, 18 patients who completed the BRIDGE study, and 69 patients who completed the BALANCE study), and 29 patients who completed the BRIGHT study in the 2 mg/kg every four weeks extension study (PB-102-F51, NCT03614234).
After the approval of Elfabrio in the U.S. and the EU, sponsorship and administration of the extension studies was transferred to Chiesi. Over time, and as Elfabrio is approved for marketing in different jurisdictions, patients switch out of the open-label extension studies. Most of them have transferred to a commercial setting; others withdraw for other reasons. Accordingly, the 1 mg/kg every two weeks dosage extension study is now closed as most patients have transferred to commercial or expanded access programs. In addition, most of the patients that enrolled in the 2 mg/kg every four weeks dosage extension study are now being treated with Elfabrio on a commercial basis.
Pediatric FLY Study
Chiesi is sponsoring, with the company’s collaborative efforts, a clinical trial entitled ‘Multi-Centre, Open-label Trial to Assess the Safety, Pharmacodynamics, Efficacy, and Pharmacokinetics of pegunigalsidase alfa in Patients From 2 Years to Less Than 18 Years of Age With Confirmed Fabry Disease’ (NCT06328608). Recruitment has commenced. The design of the study is based on the Initial Pediatric Study Plan (iPSP) agreed to with the FDA and the pediatric investigation plan (PIP) for Elfabrio, which has been accepted, as amended, by the Pediatric Committee (PDCO) of the EMA.
Japanese RISE Study
Chiesi is currently enrolling patients in its clinical trial entitled ‘A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease,’ or the RISE study (NCT05710692). The aim of the RISE study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease.
Product Development Pipeline
The company’s corporate strategy includes the development of treatments for rare and orphan diseases. To execute on the strategy, the company is turning its focus to new, early-stage product candidates that treat indications for which there are high unmet needs in terms of efficacy and safety, including renal diseases. Treatments of interest will address both genetic and non-genetic diseases. The company intends to use its ProCellEx platform and PEGylation capabilities, as well as other modalities, such as small molecules and antibodies, to take advantage of highly innovative opportunities. The company’s current pipeline of product candidates includes PEGylated uricase for the treatment of uncontrolled gout, Long Acting (LA) DNase I for the treatment of NETs, and other technologies and preclinical assets.
PEGylated Uricase (PRX-115)
PRX-115 is the company’s recombinant PEGylated uricase (urate oxidase) – a chemically modified enzyme under development for the potential treatment of patients with uncontrolled gout. The uricase enzyme, which does not exist naturally in humans, converts urate to allantoin, which is easily eliminated through urine. This recombinant enzyme, expressed via the company’s ProCellEx system, is designed to lower urate levels and improve clinical manifestation of the disease while having low immunogenicity and increased half-life of the drug in the blood. Pre-clinical data demonstrates long half-life, reduced immunogenic risk, and high specific activity supports the potential of PRX-115 to be a safe and effective treatment for patients with gout. One-month multiple dosing toxicity studies in two animal species and a six-month multiple dosing toxicity study in one animal species were conducted to support single- and multiple-dose studies in humans.
In March 2023, the company initiated its phase I clinical trial of PRX-115 for the potential treatment of uncontrolled gout entitled ‘A Double-blind, Placebo-controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Properties of PRX-115 in Adult Volunteers With Elevated Uric Acid Levels’ (NCT05745727), or the FIH study. The study was conducted at New Zealand Clinical Research (NZCR) under the New Zealand Medicines and Medical Devices Safety Authority (MedSafe) and the Health and Disability Ethics Committee (HDEC) guidelines. The study was initially designed to include seven sequential dosing cohorts, each composed of eight subjects (six active and two placebo), a 3:1 ratio. Subjects in each cohort, males and females aged 18 through 65, received a single dose of PRX-115 and were analyzed for safety, pharmacokinetics (PK), and pharmacodynamics (PD) (concentrations of plasma urate) for 85 days.
PRX-119
PRX-119 is the company’s plant cell-expressed PEGylated recombinant human DNase I product candidate, which the company is designing to have an elongated half-life in the circulation for the potential treatment of NETs-related diseases. The company’s proprietary modified DNase I, which the company has designed for long and customized systemic circulation in the bloodstream, may potentially enable effective treatment for these conditions.
Intellectual Property
The company works to continually enhance, strengthen, and protect its intellectual property and now holds a broad portfolio of approximately 75 patents globally, including in Europe, the United States, Israel, and additional countries worldwide. The company’s patents are designed to protect its proprietary technology, proprietary products, and product candidates, and their methods of use. Additionally, the company has approximately 50 pending patent applications.
During the year ended December 31, 2024, the company received the following:
New patents in each of the USA, Australia, Chile, and Mexico for the patent family named ‘Therapeutic Regimen For the Treatment of Fabry Using Stabilized Alpha-galactosidase, adding to the single previously granted patent in such family.
A new patent in Japan for the patent family named ‘Removal of Constructs from Transformed Cells.’
The company seeks to protect its competitive position by filing United States, European Union, Israeli, and other foreign patent applications covering its technology, including both new technology and improvements to existing technology.
In April 2005, Protalix Ltd. entered into a license agreement with Icon Genetics AG, or Icon, pursuant to which the company received an exclusive worldwide license to develop, test, use, and commercialize Icon’s technology to express certain proteins in its ProCellEx protein expression system.
ProCellEx is the company’s registered trademark.
Competition
With respect to Gaucher disease, the company faces competition primarily from two ERTs, Sanofi Genzyme’s Cerezyme and Takeda’s (Shire) Vpriv.
With respect to Fabry disease, the company faces competition primarily from Sanofi Genzyme (Fabrazyme), Takeda (Replagal), and Amicus (Galafold).
With respect to uncontrolled gout, the company faces competition from Horizon Therapeutics Public Limited Company (Krsytexxa).
Agreements and Partnerships
Elelyso – Pfizer
The company has licensed to Pfizer the global rights to Elelyso in all markets, excluding Brazil, pursuant to an Amended and Restated Exclusive License and Supply Agreement, or the Amended Pfizer Agreement, which the company entered into with Pfizer in October 2015 to amend and restate its initial Exclusive License and Supply Agreement with Pfizer, or the Pfizer Agreement.
Elelyso – Fundação Oswaldo Cruz (Fiocruz)
Elelyso, marketed as BioManguinhos alfataliglicerase in Brazil, is commercialized in Brazil through the Brazil Agreement with Fiocruz, which became effective in January 2014.
Elfabrio (pegunigalsidase alfa\PRX-102) – Chiesi Farmaceutici
The company has entered into two exclusive global licensing and supply agreements for PRX-102 for the treatment of Fabry disease with Chiesi; the Exclusive License and Supply Agreement dated as of October 17, 2017, made by and between Protalix Ltd. and Chiesi, or the Chiesi Ex-US Agreement, and the Exclusive License and Supply Agreement dated as of July 23, 2018, made by and between Protalix Ltd. and Chiesi, or the Chiesi US Agreement. The Chiesi Ex-US Agreement and the Chiesi US Agreement are referred to herein collectively as the Chiesi Agreements.
Manufacturing
Elelyso, Elfabrio, and the company’s drug product candidates must be manufactured in a clean environment and in compliance with cGMPs set by the FDA and other relevant foreign regulatory authorities.
Since 2017, the company’s manufacturing facility has been approved by the FDA as a multi-product facility. The company’s facility’s current capacity can serve all of its current and expected commercial and clinical needs.
The company’s manufacturing facilities are subject to inspections by various regulatory authorities from time to time. The company has undergone successful inspections by the FDA, the Irish Medicines Board (under the EMA’s centralized marketing authorization procedure), the Brazilian National Health Surveillance Agency (ANVISA), the Israeli Ministry of Health, the Turkish Ministry of Health, the Australian Therapeutic Goods Administration (TGA), and Health Canada.
Government Regulations
Any drugs for which the company receives FDA approval are subject to continuing regulation by the FDA, including, among other things, record-keeping requirements, reporting of adverse effects with the product, reporting of changes in distributed products which would require field alert reports (FARs), drugs and biological product deviation reports (BPDRs) providing the FDA with updated safety and efficacy information, product sampling and distribution requirements, complying with certain electronic records and signature requirements, and complying with FDA promotion and advertising requirements.
In addition to FDA regulations, the company is also subject to evolving federal, state, and local environmental, health, and safety laws and regulations. In the past, compliance with environmental, health, and safety laws and regulations has not had a material effect on its capital expenditures.
In the United States, the company’s activities are potentially subject to regulation by various federal, state, and local authorities in addition to the FDA, including the Centers for Medicare and Medicaid Services, other divisions of the U.S. Department of Health and Human Services (e.g., the Office of Inspector General), the U.S. Department of Justice, and individual U.S. Attorney General offices within the Department of Justice, and state and local governments. These regulations include the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA; the Health Care Reform Law; and the FFDCA.
The company used the Rolling Review option for its taliglucerase alfa NDA, which it completed in April 2010.
Depending upon the timing, duration, and specifics of FDA marketing approval of the company’s product candidates, some of its U.S. patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, commonly referred to as the Hatch-Waxman Amendments.
Research and Development
For the year ended December 31, 2024, the company’s total research and development expenses were approximately $13.0 million.
History
Protalix BioTherapeutics, Inc. was founded in 1993. The company was incorporated in 1993.
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