The drug is manufactured and marketed in Israel by Protalix Biotherapeutics but licensed under the name Elelyso in the rest of the world by Pfizer. The Israeli firm has amended its marketing deal with the Pharma Giant in order to enter a supply and technology transfer agreement with Fundação Oswaldo Cruz (known as Fiocruz), an arm of the Brazilian Ministry of Health.
“This technology transfer deal is unique to the Brazilian market,” Kari Watson, a spokesperson for Protalix, told in-Pharmatechnologist.com. “The intention is to build the capacity and skills required for the Brazilian government to produce a sustainable, high quality, and cost effective supply of UPLYSO in Brazil.”
Fiocruz has committed to purchasing $40m of the drug during the first two years, and then a further $40m per year until payment reaches $280m when the technology transfer will be completed.
Once this is done, the Government will be the sole source of this treatment option for Gaucher’s patients in Brazil. The country has approximately 600 patients diagnosed with the disease, Watson added, which represents the third-largest market in the world.
As part of the deal, Pfizer will relinquish its commercial rights to Uplyso in Brazil once the Brazilian National Health Surveillance Agency (ANVISA) approves both the plant being built next year and Fiocruz’ registration of the drug. Pfizer will then receive a maximum of $12.5m a year from profits generated by the drug.
Plant That Uses Plants
According to Fiocruz’ website, the manufacturing facility being constructed solely for the production of Uplyso in Brazil will be fully operational in the city of Eusebio from 2016 and will use Protalix’ Procellex technology platform that produces biologics from plant cells.
Instead of traditional mammalian or microbial cultures, the Israeli firm uses such cells as carrots or tobacco to produce a variety of proteins including antibodies, complex enzymes, and plant-derived pharmaceuticals.
So far the only approved plant-based manufactured drug is Uplyso - approved in the US, Brazil, Israel and Uruguay - though Protalix has spoken previously on using this regulatory thumbs-up as a springboard to launch its other projects, which also use the Procellex technology.
According to Fiocruz, “the technology ensures higher security than the biological produced from viruses and bacteria” and produces fewer side effects and less resistance from the human body. “Moreover,” says the website, “it requires less investment.”
Gaucher's Disease Treatments
Genzyme, as a major player in the treatment of Gaucher’s Disease, has seen a great deal of success with its drug Cerezyme, partly due to its average launch price of $200,000 a year, which made it one of the world’s most expensive drugs.
Protalix has priced Uplyso at a quarter of this, according to the company’s CEO David Aviezer speaking in February.
The third player is Shire, whose velaglucerase alfa has orphan marketing status and exclusivity in the EU until 2020, following a ruling by the European Commission last year.