Protalix BioTherapeutics has announced that Fundação Oswaldo Cruz (Fiocruz), an arm of the Brazilian Ministry of Health, has issued a letter of intent to purchase significant quantities of Uplyso (alfataliglicerase) to treat people with Gaucher disease in Brazil.
Known as Elelyso (taliglucerase alfa) outside of Latin America, the treatment was first approved by the U.S. Food and Drug Administration for adults with Type 1 Gaucher disease in 2012, and was approved for pediatric use in 2014. Since then, many other countries have also approved of its use as a Gaucher disease treatment.
Alfataliglicerase is the first plant cell-based enzyme replacement therapy (ERT) for Gaucher disease. It is also the first approved plant cell-expressed drug that derives from ProCellEx, Protalix’s cell-based protein manufacturing system using genetically modified carrot cells.
Gaucher disease is a rare lysosomal storage disorder, and alfataliglicerase is a form of the human lysosomal enzyme, glucocerebrosidase.
The Brazilian National Health Surveillance Agency, known as ANVISA, granted regulatory approval for Uplyso to treat adults with Gaucher disease in March 2013, and extended that approval to children last month.
The letter requests three shipments of Uplyso: the first of which is to be delivered by mid-2017, and the last to be delivered by year’s end. The company expects to claim about $24 million in revenue in the sale.
“To date, we have sold relatively small quantities of alfataliglicerase to the Brazilian Ministry of Health, which has allowed physicians and patients in Brazil to experience firsthand the drug’s beneficial efficacy and safety profile,” Moshe Manor, president and chief executive officer of Protalix, said in a press release. “The Brazilian Ministry’s request to purchase considerably larger quantities comes on the heels of advanced negotiations … regarding the potential of alfataliglicerase becoming the preferred enzyme replacement therapy for the approximately 700 Gaucher patients treated in Brazil.”
Common adverse reactions for this medication include itching, headaches, nausea, and joint pain.
Protalix has licensed global development and commercialization rights for alfataliglicerase to Pfizer, except for Brazil, where Protalix retains full rights. Brazil has the third largest number of Gaucher patients identified worldwide, after the U.S. and Israel.