Literature DB >> 26149580

Molecular pharming's foot in the FDA's door: Protalix's trailblazing story.

Tsafrir S Mor1.   

Abstract

OBJECTIVES: This short commentary examines the factors that led to Food and Drug Administration's approval of the first plant-derived biologic.
RESULTS: In 2012, the first plant-derived protein pharmaceutical (biologic) was approved for commercial use in humans. The product, a recombinant form of human β-glucocerebrosidase marketed as ELELYSO, was developed by Protalix Biotherapeutics (Carmiel, Israel). The foresight to select this particular therapeutic product for development, flawless production pipeline, and serendipity seem to provide the key in explaining how ELELYSO became the first plant-derived biologic to achieve approval by Food and Drug Administration.
CONCLUSIONS: While the circumstances that enabled Protalix and its scientists to become the first to arrive at this historic milestone are perhaps unique, it is anticipated that more biologics will follow suit in winning regulatory endorsement.

Entities:  

Keywords:  Ebola; FDA-approval; Gaucher’s disease; Molecular pharming; Plant-derived biologics

Mesh:

Substances:

Year:  2015        PMID: 26149580      PMCID: PMC4583803          DOI: 10.1007/s10529-015-1908-z

Source DB:  PubMed          Journal:  Biotechnol Lett        ISSN: 0141-5492            Impact factor:   2.461


  22 in total

1.  Expression of hepatitis B surface antigen in transgenic plants.

Authors:  H S Mason; D M Lam; C J Arntzen
Journal:  Proc Natl Acad Sci U S A       Date:  1992-12-15       Impact factor: 11.205

2.  Clinical pharmacology as a cornerstone of orphan drug development.

Authors:  Edward D Bashaw; Shiew-Mei Huang; Timothy R Coté; Anne R Pariser; Christine E Garnett; Gilbert Burckart; Lei Zhang; Angela Y Men; Christine D Le; Rosane Charlab; Jogaro V Gobburu; Lawrence J Lesko
Journal:  Nat Rev Drug Discov       Date:  2011-10-31       Impact factor: 84.694

3.  Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease.

Authors:  Ari Zimran; Einat Brill-Almon; Raul Chertkoff; Milan Petakov; Francisco Blanco-Favela; Eduardo Terreros Muñoz; Sergio E Solorio-Meza; Dominick Amato; Gloria Duran; Fiorina Giona; Rene Heitner; Hanna Rosenbaum; Pilar Giraldo; Atul Mehta; Glen Park; Mici Phillips; Deborah Elstein; Gheona Altarescu; Mali Szleifer; Sharon Hashmueli; David Aviezer
Journal:  Blood       Date:  2011-09-06       Impact factor: 22.113

Review 4.  Gaucher disease and Fabry disease: new markers and insights in pathophysiology for two distinct glycosphingolipidoses.

Authors:  Maria J Ferraz; Wouter W Kallemeijn; Mina Mirzaian; Daniela Herrera Moro; Andre Marques; Patrick Wisse; Rolf G Boot; Lianne I Willems; H S Overkleeft; J M Aerts
Journal:  Biochim Biophys Acta       Date:  2013-11-13

Review 5.  Sphingolipid lysosomal storage disorders.

Authors:  Frances M Platt
Journal:  Nature       Date:  2014-06-05       Impact factor: 49.962

6.  Production of glucocerebrosidase with terminal mannose glycans for enzyme replacement therapy of Gaucher's disease using a plant cell system.

Authors:  Yoseph Shaaltiel; Daniel Bartfeld; Sharon Hashmueli; Gideon Baum; Einat Brill-Almon; Gad Galili; Orly Dym; Swetlana A Boldin-Adamsky; Israel Silman; Joel L Sussman; Anthony H Futerman; David Aviezer
Journal:  Plant Biotechnol J       Date:  2007-05-24       Impact factor: 9.803

Review 7.  Plant molecular pharming for the treatment of chronic and infectious diseases.

Authors:  Eva Stoger; Rainer Fischer; Maurice Moloney; Julian K-C Ma
Journal:  Annu Rev Plant Biol       Date:  2014-02-24       Impact factor: 26.379

8.  Gaucher disease: clinical profile and therapeutic developments.

Authors:  Timothy M Cox
Journal:  Biologics       Date:  2010-12-06

9.  Glycosylation and functionality of recombinant β-glucocerebrosidase from various production systems.

Authors:  Yoram Tekoah; Salit Tzaban; Tali Kizhner; Mariana Hainrichson; Anna Gantman; Myriam Golembo; David Aviezer; Yoseph Shaaltiel
Journal:  Biosci Rep       Date:  2013-09-25       Impact factor: 3.840

10.  Reversion of advanced Ebola virus disease in nonhuman primates with ZMapp.

Authors:  Xiangguo Qiu; Gary Wong; Jonathan Audet; Alexander Bello; Lisa Fernando; Judie B Alimonti; Hugues Fausther-Bovendo; Haiyan Wei; Jenna Aviles; Ernie Hiatt; Ashley Johnson; Josh Morton; Kelsi Swope; Ognian Bohorov; Natasha Bohorova; Charles Goodman; Do Kim; Michael H Pauly; Jesus Velasco; James Pettitt; Gene G Olinger; Kevin Whaley; Bianli Xu; James E Strong; Larry Zeitlin; Gary P Kobinger
Journal:  Nature       Date:  2014-08-29       Impact factor: 49.962

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  17 in total

1.  Glyco-Engineering of Plant-Based Expression Systems.

Authors:  Rainer Fischer; Tanja Holland; Markus Sack; Stefan Schillberg; Eva Stoger; Richard M Twyman; Johannes F Buyel
Journal:  Adv Biochem Eng Biotechnol       Date:  2021       Impact factor: 2.635

2.  A plant-derived cocaine hydrolase prevents cocaine overdose lethality and attenuates cocaine-induced drug seeking behavior.

Authors:  Katherine E Larrimore; Latha Kannan; R Player Kendle; Tameem Jamal; Matthew Barcus; Kathryn Stefanko; Jacquelyn Kilbourne; Stephen Brimijoin; Chang-Guo Zhan; Janet Neisewander; Tsafrir S Mor
Journal:  Prog Neuropsychopharmacol Biol Psychiatry       Date:  2020-05-06       Impact factor: 5.067

3.  Production of Recombinant Proteins in Transgenic Tobacco Plants.

Authors:  Tim H Szeto; Pascal M W Drake; Audrey Y-H Teh; Nicole Falci Finardi; Ashleigh G Clegg; Mathew J Paul; Rajko Reljic; Julian K-C Ma
Journal:  Methods Mol Biol       Date:  2022

Review 4.  Quality Control and Downstream Processing of Therapeutic Enzymes.

Authors:  David Gervais
Journal:  Adv Exp Med Biol       Date:  2019       Impact factor: 2.622

5.  Technoeconomic analysis of semicontinuous bioreactor production of biopharmaceuticals in transgenic rice cell suspension cultures.

Authors:  Jasmine M Corbin; Matthew J McNulty; Kantharakorn Macharoen; Karen A McDonald; Somen Nandi
Journal:  Biotechnol Bioeng       Date:  2020-07-14       Impact factor: 4.395

6.  Plant expression systems, a budding way to confront chikungunya and Zika in developing countries?

Authors:  Jaime A Cardona-Ospina; Juan C Sepúlveda-Arias; L Mancilla; Luis G Gutierrez-López
Journal:  F1000Res       Date:  2016-08-31

7.  Plant-expressed cocaine hydrolase variants of butyrylcholinesterase exhibit altered allosteric effects of cholinesterase activity and increased inhibitor sensitivity.

Authors:  Katherine E Larrimore; I Can Kazan; Latha Kannan; R Player Kendle; Tameem Jamal; Matthew Barcus; Ashini Bolia; Stephen Brimijoin; Chang-Guo Zhan; S Banu Ozkan; Tsafrir S Mor
Journal:  Sci Rep       Date:  2017-09-05       Impact factor: 4.379

8.  A transatlantic perspective on 20 emerging issues in biological engineering.

Authors:  Bonnie C Wintle; Christian R Boehm; Catherine Rhodes; Jennifer C Molloy; Piers Millett; Laura Adam; Rainer Breitling; Rob Carlson; Rocco Casagrande; Malcolm Dando; Robert Doubleday; Eric Drexler; Brett Edwards; Tom Ellis; Nicholas G Evans; Richard Hammond; Jim Haseloff; Linda Kahl; Todd Kuiken; Benjamin R Lichman; Colette A Matthewman; Johnathan A Napier; Seán S ÓhÉigeartaigh; Nicola J Patron; Edward Perello; Philip Shapira; Joyce Tait; Eriko Takano; William J Sutherland
Journal:  Elife       Date:  2017-11-14       Impact factor: 8.140

9.  Human Indoleamine 2,3-dioxygenase 1 (IDO1) Expressed in Plant Cells Induces Kynurenine Production.

Authors:  Michele Bellucci; Andrea Pompa; Carine De Marcos Lousa; Eleonora Panfili; Elena Orecchini; Elisa Maricchiolo; Daniele Fraternale; Ciriana Orabona; Francesca De Marchis; Maria Teresa Pallotta
Journal:  Int J Mol Sci       Date:  2021-05-12       Impact factor: 5.923

10.  Depth Filters Containing Diatomite Achieve More Efficient Particle Retention than Filters Solely Containing Cellulose Fibers.

Authors:  Johannes F Buyel; Hannah M Gruchow; Rainer Fischer
Journal:  Front Plant Sci       Date:  2015-12-21       Impact factor: 5.753

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