This week, the US Food and Drug Administration announced their approval of the first ever drug produced in a genetically engineered plant cell.
The drug, Elelyso (taliglucerase alfa), is an enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder that disproportionately affects Ashkenazi Jews.
People with the disease don’t produce enough of an enzyme called glucocerebrosidase, and this deficiency causes fatty materials to collect in the the spleen, liver, and kidneys – resulting in organ damage, anemia, low blood platelet counts, and bone problems.
Scientists at the Israeli biotechnology company Protalix Biotherapeutics developed a method to create the human enzyme that these patients lack in carrot cells by inserting a gene that encodes the protein into the cells.
Patients treated with the resulting enzyme in clinical trials fared at least as well as those given a similar drug, Cerezyme. The plant-made drug reduced spleen volume by an average of 40%.
The new option could help prevent drug shortages, such as those that occurred as recently as 2009 and 2011. Nature reports:
Manufacturers of the two other Gaucher drugs – Genzyme in the US and Shire in Ireland – produce their therapeutic enzymes in mammalian cells. Structurally, Elelyso resembles Genzyme’s Cerezyme, but it’s cheaper to produce because of the high maintenance that animal-cell cultures require.
Additionally, viruses and other pathogens that contaminate animal stocks don’t threaten plant-cell cultures.
For the average patient, Cerezyme can cost $200,000 per person per year. Elelyso will cost about 25% less than Cerezyme. And 60% of the profits from US sales will go to Pfizer, which made a deal with Protalix in 2009.
[Via Nature News]
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