First plant-made biologic approved

Carrot cell bioreactors

The US Food and Drug Administration in May approved Elelyso (taliglucerase alfa), an enzyme produced in genetically engineered carrot cells, for treating type 1 Gaucher's disease. This is the first plant-made drug approved by the regulators, and for Israeli company Protalix BioTherapeutics of Carmiel, it is the first product made in their ProCellEx protein expression system to reach the market. The plant cell platform produces recombinant proteins with a glycan and amino acid structure similar to naturally produced human counterparts. Some 10,000 patients worldwide have Gaucher's, a rare genetic disorder in which individuals fail to produce the enzyme glucocerebrosidase. Elelyso, a recombinant form of human glucocerebrosidase, which is injectable, replaces that enzyme and thus prevents lipids from accumulating in organs and tissues, eventually damaging the liver and spleen, leading to low red blood cell and platelet counts, and bone problems. Protalix and US partner Pfizer of New York are pricing Elelyso at a 25% discount from Genzyme's Cerezyme (imiglucerase), the market leader, a price they hope will persuade clinicians and patients to switch. They are also touting this platform for its dependability, pointing to a year-long spate of supply disruptions for comparable enzymes made by Genzyme, wholly owned by Paris-based Sanofi, to treat Gaucher's disease and Fabry's disease (Nat. Biotechnol. 28, 994, 2010). Pfizer and partner are attempting to position Elelyso as the most reliable product on the market launching a “supply continuity program,” whereby a 24 months' supply is maintained. They have also developed several programs to help individuals with Gaucher's disease to afford treatments with this new product. It may be too late, however, to gain a significant market share. A year ago, at the height of the supply disruptions affecting Genzyme's enzyme production, the Protalix-Pfizer product stood to gain a majority share, but regulatory delays in approving the plant-based product meant that that opportunity was lost. In a separate development earlier this year, Cambridge, Massachusetts–based Genzyme, a Sanofi company, announced four-year data from a phase 2 clinical trial for an orally administered version of its treatment for type 1 Gaucher's disease, eliglustat tartrate. Ireland-based Shire also has a competing injectable product for treating Gaucher's disease, called Vpriv (velaglucerase alfa).

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Fox, J. First plant-made biologic approved. Nat Biotechnol 30, 472 (2012). https://doi.org/10.1038/nbt0612-472

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