This website is intended for healthcare professionals outside of the US and UK
Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). Heterozygous females were not studied in the clinical trials.1 Hunter syndrome patients have mutations in the IDS gene which encodes iduronate-2-sulfatase (I2S), an enzyme necessary for the break-down of mucopolysaccharides (glycosaminoglycans (GAGs)). Without the I2S enzyme, GAGs accumulate in the cells, tissues and organs of MPS II patients resulting in a variety of symptoms associated with Hunter syndrome.2 A formulation of I2S, Elaprase is produced by recombinant DNA technology in a human cell line, and provides a disease specific form of enzyme replacement therapy (ERT).3 For more than a decade, Elaprase has provided patients with a treatment option that has been shown to improve many of the somatic signs and symptoms of Hunter syndrome.
Where is Elaprase
Elaprase is currently available for the treatment of Hunter syndrome patients in:
Albania, Algeria, Argentina, Australia, Bahrain, Belarus, Brazil, Canada, Chile, Chile, Colombia, Ecuador, European Union, Guatemala, Hong Kong, Indonesia, Israel, Japan, Kazakhstan, Kuwait, Macedonia, Malaysia, Mexico, Morocco, New Zealand, Oman, Panama, Paraguay, Peru, Philippines, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Africa, South Korea, Switzerland, Taiwan, Thailand, Turkey, Ukraine, United Arab Emirates, Uruguay, Venezuela, Vietnam.
The Hunter Outcome Survey
The Hunter Outcome Survey (HOS) was established as a long-term, open-ended, multicentre, global registry designed to collect information on patients with Hunter syndrome and to address the post-approval commitments of Elaprase. The HOS has been collecting real-world data on the clinical presentation and progression of Hunter syndrome during routine assessments for over ten years. The objectives of this registry are to gather information on the effectiveness and safety of Elaprase treatment and to clarify the disease progression in MPS II patients.4
10 years of Elaprase
An analysis was carried out to compare the survival data of Elaprase treated and untreated patients identified that Elaprase treatment significantly improved the survival of MPS II patients by an average of 11.8 years and reduced the risk of mortality by 54%.5
As of July 2016, 1200 patients, from 134 clinics, in 33 countries had been enrolled in the HOS, making it the largest global source of data on Hunter syndrome.5
The shelf-life of Elaprase was extended from 2 years to 3 years as demonstrated by the SmPC update.1
Elaprase cited as ERT treatment for Hunter syndrome in guidelines for Latin America.6
HOS study results focusing on the effect of Elaprase in MPS II growth rates were published.7
The publication of the expert panel consensus on the use of Elaprase treatment in severe MPS II patients.8
Elaprase cited as ERT for treatment of Hunter syndrome in European Guidelines.9
An analysis of HOS data on the safety and effectiveness of Elaprase use in patients younger than 6 years old found Elaprase had a similar safety profile in these patients permitting the early initiation of treatment in MPS II patients, which is important for treating this progressive disease.10
Analysis of HOS data confirmed that, taking into consideration appropriate factors, it is feasible for Hunter syndrome patients, including those who are severely affected, to receive ERT infusions at home.11
A report on the mortality and causes of death of untreated Hunter syndrome patients was published.12
The initial report from the HOS was published after following 263 Hunter syndrome patients.4
Elaprase received approval in Europe in 2007.13
Elaprase received FDA approval in the USA.14
The HOS is set up to gather data on Hunter syndrome patients receiving ERT.4
Elaprase Summary of Product Characteristics. 2018. Last accessed August 2018.
Neufeld EF, Muenzer J. The mucopolysaccharidoses. In Scriver CR, Beaudet Al, Sly WS, Valle D (eds) The metabollic and molecular bases of inherited disease. McGraw-Hill, New York. 2001;3421-3452.
Muenzer J, Beck M, et al. Long-term, open-label extension study of idursulfase in the treatment of Hunter syndrome. Genet Med. 2011;13:95-101.
Wraith JE, Beck M, et al. Initial report from the Hunter Outcome Survey. Genet Med. 2008;10:508-516.
Burton BK, Jego V, et al. Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS). J Inherit Metab Dis. 2017; 40(6):867-874.
Giugliani R, Villarreal MLS, et al. Guidelines for diagnosis and treatment of Hunter syndrome for clinicians in Latin America. Genet Mol Biol. 2014; 37(2):315-329.
Jones SA, Parini R, et al. The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS). Mol Genet Metab. 2013; 109(1):41-48.
Muenzer J, Bodamer O, et al. The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus. Eur J Pediatr. 2012;171(1):181-188.
Scarpa M, Almassy Z, et al. Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease. Orphanet Journal of Rare Diseases. 2011;6:72.
Muenzer J, Beck M, et al. Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome Survey. Genet Med. 2011;13(2):102-9.
Burton BK, Guffon N, et al. Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis – data from the Hunter Outcome Survey. Mol Genet Metab. 2010;101(2-3):123-9.
Jones SA, Almassy Z, et al. Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS). J inherit Metab Dis. 2009;32(4):534-543.
EMA EPAR. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/000700/WC500023003.pdf (Last accessed January 2018).
Food and Drug Administration. Application 125151/0 Approval Letter. (Last accessed January 2018)