ELAPRASE

Back

ELAPRASE® treatment
has been shown to increase survival

This website is intended for healthcare professionals outside of the US and UK

Real-world evidence (RWE) shows ELAPRASE has a positive effect on survival in MPS II Ipatients and in a number of somatic parameters over the
long-term.1–14 In addition, RWE shows ELAPRASE is well tolerated, typically without any need for treatment interruption;15–17 and RWE supports that timely diagnosis and early ELAPRASE initiation are key to improving clinical outcomes due to the progressive and irreversible nature of MPS II.18–20

ELAPRASE is supported by over 15 years of real-world evidence including data from the Hunter Outcome Survey (HOS) patient registry.12,14 Since the launch in 2005, the HOS, which is a multi-centre, multinational observational study, has collected real-world data on the natural history of Hunter syndrome and the safety and effectiveness of using ELAPRASE in management from more than 1000 patients:1

In July 2016, >1000 patients were enrolled in the HOS

> 1000 patients

The >1000 patients enrolled in the HOS were from 134 centres

134 centres

The 134 centres with patients in the HOS were from 33 countries

33 countries


ELAPRASE INCREASED SURVIVAL of Hunter syndrome patients:a

graph-1-layer-1 graph-1-layer-2 graph-1-layer-3
graph-1-layer-4
number-of-paients-layer-1
number-of-paients-layer-2

Kaplan–Meier survival analysis showing median survival in MPS II ELAPRASE-treated and untreated patients was 33.0 years and 21.2 years respectively.

Calculate the increased survival with ELAPRASE

Discover the result

ELAPRASE treatment improved patient survival by

11.8 years

compared to untreated patients

ELAPRASE REDUCED THE RISK OF DEATH of Hunter syndrome patients:a

graph-2-layer-1 graph-2-layer-2 graph-2-layer-3
graph-2-layer-4

Direct adjusted survival curves of predicted values for the risk of death for ELAPRASE-treated and untreated patients based on the primary Cox regression model.

Calculate the reduced risk of death with ELAPRASE

Discover the result

ELAPRASE-treated patients had a

54% (HR=0.46)

lower risk of death compared with untreated patients


ELAPRASE reduced and stabilised LVMI over 3 years.2,3

  • 3-year RWE from HOS study: after 3 years of ELAPRASE treatment, median percentage change in LVMI was -9.3% (n=52).2,b

ELAPRASE treatment significantly reduced and sustained uGAG levels over 2+ years.2–5

  • 3-year RWE from HOS study: over 3 years of ELAPRASE treatment, the absolute change in median uGAG levels was –177.8 μg/mg creatinine (n=83).2,b

ELAPRASE significantly reduced liver and spleen size over 3+ years.2,3

  • 3-year RWE from HOS study: after 3 years of ELAPRASE treatment, the reduction in median liver size was 54% (n=53) and the reduction in median spleen size was 33% (n=17).2,b

ELAPRASE provided sustained improvements in pulmonary function (FVC and FEV1) over 3 years.2

  • 3-year RWE from HOS study: after 3 years of ELAPRASE treatment, the median percentage change in FVC was 29.7% (n=23), and in FEV1 was 22.8% (n=22).2,b

ELAPRASE increased 6MWT distance over 3 years indicating an improvement in physical functioning.2

  • 3-year RWE from HOS study: after 3 years of ELAPRASE treatment, the median percentage change in 6MWT distance was 10.6% (n=26).2,b

ELAPRASE provided improvements in growth rate.6–8

RWE supports improvement in joint range of motion (JROM) with ELAPRASE, as seen in the pivotal extension trial.8,10

Improvements in HRQoL with ELAPRASE seen in the pivotal extension trial and is supported by RWE.3,10,11

References:

  1. Burton B.K, Jego V, Mikl J et al. J Inherit Metab Dis. 2017;40(6):867-874.

  2. Muenzer J, Beck M, Giugliani R et al. Orphanet J Rare Dis 2017;12:161.

  3. Parini R, Rigoldi M, Tedesco L et al. Mol Genet Metab Rep. 2015;3:65-74.

  4. Tomanin R, Zanetti A, D'Avanzo F et al. Orphanet J Rare Dis 2014;9:129.

  5. Serra J, Miñana I.V, Fernández R.C et al. Med Clin (Barc). 2015;145(9):392-398.

  6. Parini R, Jones SA, Harmatz PR et al. Mol Genet Metab. 2016;117(4):438-446.

  7. Jones SA, Parini R, Harmatz P et al. Mol Genet Metab. 2013;109(1):41-48.

  8. Giugliani R, Hwu WL, Tylki-Szymanska A et al. Genet Med. 2014;16(6):435-441.

  9. Takeda data on file, 2018 [HOS Annual Report (EMA)].

  10. Muenzer J, Beck M, Eng CM et al. Genet Med. 2011a;13(2):95-101.

  11. Guffon N, Heron B, Chabrol B et al. Orphanet J Rare Dis. 2015;10:43.

  12. Whiteman DA and Kimura A. Drug Des Devel Ther. 2017;11:2467-2480.

  13. Data on File (ELAPRASE) July 2020.

  14. Muenzer J, Simon A J, Tylki-Szymanska A et al. Orphanet J Rare Dis. 2017;12(1):82.

  15. Burton BK, Whiteman DA and HOS Investigators. Mol Genet Metab. 2011;103(2):113-120.

  16. Barbier AJ, Bielefeld B, Whiteman DA et al. Mol Genet Metab. 2013;110(3):303-310.

  17. Pano A., Barbier AJ, Bielefeld B et al. Orphanet J Rare Dis. 2015;10:50.

  18. Tajima G, Sakura N, Kosuga M et al. Mol Genet Metab. 2013;108:172-177.

  19. Lampe C, Atherton A, Burton BK et al. JIMD Rep. 2014;14:99-113.

  20. Tylki-Szymanska A, Jurecka A, Zuber Z et al. Acta Paediatr 2012;101:e42–e47.

calculator