Protalix BioTherapeutics, a biopharma specializing in the development of recombinant therapeutic proteins, announced that the Brazilian National Health Surveillance Agency, known as ANVISA, has granted regulatory approval for Uplyso (alfataliglicerase) to treat Gaucher disease in children.
ANVISA approved Uplyso in March 2013 for long-term enzyme replacement therapy (ERT) in adults with a confirmed diagnosis of Type 1 Gaucher disease.
Now, Uplyso is also approved as a long-term ERT in children age 4 and older with the disease.
Uplyso, known as Elelyso (taliglucerase alfa) outside of Latin America, was first approved in 2012 by the U.S. FDA for adults with Type 1 Gaucher disease, and in 2014 for pediatric use. Many other countries have also approved the drug for Gaucher disease.
Protalix licensed the worldwide development and commercialization rights for taliglucerase alfa to Pfizer — except for Brazil, where Protalix still retains full rights.
"ANVISA’s approval of a pediatric indication for alfataliglicerase is an important milestone for Protalix in our commitment to the Gaucher community,” Moshe Manor, president and CEO of Protalix, said in a press release.
The approval supports ongoing discussions with the Brazilian Ministry of Health for the supply of a significant amount of Uplyso for people with Gaucher disease in Brazil.
“It is also an important element in our ongoing advanced negotiations with the Brazilian Ministry of Health, as alfataliglicerase is now approved for the majority of Gaucher patients in Brazil. It is estimated that pediatric patients represent approximately 30 percent of the total Gaucher patient population in Brazil,” Manor said.
Gaucher disease is a rare lysosomal storage disorder with approximately 700 known cases in Brazil – the third largest number of identified Gaucher patients worldwide after the United States and Israel. Gaucher is estimated to affect up to one in 40,000 live births worldwide.
Like any intravenous (IV) protein medicine, Uplyso may cause infusion-related reactions. The most commonly observed reactions in adults include headaches, hypersensitivity, dizziness, nausea, pruritus (itching), or localized pain.
Before considering Uplyso, Protalix advises all patients to ask for specialized information from their physician (read full prescribing information here).
ANVISA’s report is available in Portuguese here.