|Trade names||Elelyso, Uplyso, others|
|Main uses||Gaucher's disease|
|Side effects||Headache, joint pain, tiredness, nausea, itchiness, rash|
|Typical dose||60 units/kg|
|US NLM||Taliglucerase alfa|
|Elimination half-life||18.9-28.7 minutes|
|Chemical and physical data|
|Molar mass||56638.78 g·mol−1|
Taliglucerase alfa, sold under the brand name Elelyso among others, is a medication used to treat Gaucher's disease type I. It is given by gradual injection into a vein. It is used long term.
Common side effects include headache, joint pain, tiredness, nausea, itchiness, and rash. Other side effects may include anaphylaxis. It is a glucocerebrosidase made by recombinant DNA technology and works by replacing this missing enzyme.
Taliglucerase alfa was approved for medical use in the United States in 2012. In Europe it received an orphan designation in 2010 but was declined approval in 2012 due to the similar product velaglucerase alfa having market exclusivity. In the United States it costs about 18,000 USD per dose for a 70 kg person (465,000 USD per year). It is the first plant-made medication to be approved in the United States.
It is given at a dose of 60 units per kg every two weeks. The typical dose for a 70 kg person is thus 4,200 units.
Each vial has 200 units of taliglucerase alfa.
The U.S. FDA New Drug Application (NDA) was granted approval in May 2012, for use in adults. The U.S. FDA Supplemental New Drug Application (sNDA) for pediatric use was granted approved in August 2014. In Israel, the Israeli Ministry of Health granted approval in September 2012. In Brazil, the Brazilian Health Surveillance Agency (ANVISA) granted approval in March 2013. In Canada, Health Canada issued a Notice of Compliance in May 2014, for both adults and pediatric patients.
Society and culture
For 2016, Elelyso was ranked third for pharmaceuticals with the highest cost-per-patient, with an average cost of $483,242 per year.
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