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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
ELAPRASE mode of action
Hunter syndrome is caused by the absence or deficiency of iduronate-2-sulfatase (I2S), an enzyme involved in glycosaminoglycan (GAG) catabolism.1 Pathological lysosomal storage of GAGs leads to progressive damage and dysfunction in cells, tissues and organs.1 ELAPRASE, a formulation of I2S, is produced by recombinant DNA technology in a human cell line, and provides a disease-specific form of enzyme replacement therapy (ERT).1 Click here for more information about the ELAPRASE mode of action.
ELAPRASE as standard of care
ELAPRASE, with 15 years of real-world evidence (RWE), is the gold standard treatment for Hunter syndrome patients because it can improve somatic parameters, has an established safety profile, and can increase survival. ELAPRASE is not expected to impact the central nervous system (CNS).1–6 ELAPRASE is recommended by clinical guidelines as the standard of care for patients with Hunter syndrome to be initiated as early as possible after diagnosis.7,8
Where has ELAPRASE received
ELAPRASE has received marketing authorisation in these, and additional, countries:
Albania, Algeria, Argentina, Australia, Bahrain, Belarus, Brazil, Canada, Chile, Colombia, Ecuador, European Union/EEA (Austria, Belgium, Bulgaria, Croatia, Republic of Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Ireland, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Poland, Portugal, Romania, Slovakia, Slovenia, Spain, Sweden, Iceland, Liechtenstein and Norway), Guatemala, Hong Kong, India, Indonesia, Israel, Japan, Kazakhstan, Kuwait, Malaysia, Mexico, Morocco, New Zealand, Oman, Panama, Paraguay, Peru, Philippines, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Africa, South Korea, Switzerland, Taiwan, Thailand, Turkey, UK, Ukraine, United Arab Emirates, United States, Uruguay, Venezuela, Vietnam.
15 years of ELAPRASE
ELAPRASE is the first and only ERT therapy with 15 years'
clinical experience, approved for the treatment of Hunter syndrome. The Hunter Outcome Survey (HOS) patient registry* has been collecting real-world data on the clinical presentation and progression of Hunter syndrome treated with ELAPRASE during routine assessments for over 15 years.5,7,9
An analysis was carried out to compare the survival data of ELAPRASE in treated and untreated patients, and 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 documented in a revision of the SmPC.1
ELAPRASE was cited as ERT treatment for Hunter syndrome in guidelines for Latin America.8
HOS study results focusing on the effect of ELAPRASE on MPS II growth rates were published.11
An expert panel of physicians experienced with the treatment of MPS II published a consensus document on the use of ERT in severe Hunter syndrome.11
ELAPRASE cited as ERT for treatment of Hunter syndrome in European Guidelines.9
An analysis of HOS data on the safety of ELAPRASE in patients younger than 6 years old showed a similar safety profile to that of patients aged 6 years or older when initiating treatment, therefore demonstrating the safety of early initiation of treatment in patients with MPS II. Early treatment is important for treating this progressive disease.3,12
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.5
A report on the mortality and causes of death of untreated Hunter syndrome patients was published.13
The initial report on patient enrollment in the HOS was published, with 263 patients from 16 countries participating.10
ELAPRASE received EMA approval in Europe in 2007.14
ELAPRASE received FDA approval in the USA.15
The HOS was initiated to gather data on Hunter syndrome patients receiving ERT.10
*The Hunter Outcome Survey (HOS) was established in 2005 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 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.5
Elaprase Summary of Product Characteristics. 2020.
Muenzer J, Wraith JE, Beck M et al. Genet Med. 2006;8(8):465–473
Muenzer J, Beck M, Eng CM et al. Genet Med. 2011;13(2):102-109.
Giugliani R, Hwu WL., Tylki-Szymanska A et al. Genet Med.2014;16(6):435–441.
Burton BK, Jego V, Mikl J et al. J Inherit Metab Dis. 2017;40(6):867-874.
Data on File (ELAPRASE) July 2020.
Scarpa M, Almassy Z, Beck M et al. Orphanet Journal of Rare Diseases. 2011;6:72.
Giugliani R, Villarreal ML, Valdez CA et al. Genet Mol Biol. 2014;37(2):315–329.
Whiteman DA and Kimura A. Drug Des Devel Ther 2017;11:2467-2480.
Wraith JE, Beck M, Giugliani R et al. Genet Med. 2008;10:508-516.
Jones SA, Parini R, Harmatz P et al. Mol Genet Metab. 2013;109(1):41-48.
Muenzer J, Bodamer O, Burton B et al. Eur J Pediatr. 2012;171(1):181-188.
Jones SA, Almassy Z, Beck M et al. 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.