ELAPRASE

Back

CLINICAL TRIAL EFFICACY
AND SAFETY DATA

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

ELAPRASE® clinical trials demonstrated improvements in the somatic parameters in MPS II patients.1–3 Clinical trials also demonstrated ELAPRASE is effective in treating paediatric MPS II patients.3,4 In addition, ELAPRASE has a well-established safety profile enabling long-term compliance.1–4

ELAPRASE met the primary endpoint in the pivotal trial demonstrating improvement in a composite of somatic parameters (6MWT and %FVC) in MPS II patients.1–3,a​

ELAPRASE sustained pulmonary function for 3 years1–2

The pivotal extension trial with MPSII patients aged 5–31 years:a

  • ELAPRASE provided significant and sustained improvements in absolute forced vital capacity vs baseline (absolute FVC: 1.18L baseline, mean 25.1% increase over 3 years, p<0.05).1,2

  • ELAPRASE sustained the percentage of predicted forced vital capacity (%FVC) over 3 years.1,2

The effect of ELAPRASE on percentage predicted forced vital capacity:2

pulmonary-layer-1 pulmonary-layer-2 pulmonary-layer-3
pulmonary-layer-4
Adapted by permission from the Copyright Clearance Center: Springer, Genetics in Medicine, Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome, Muenzer et al., 2010.

ELAPRASE stabilised left ventricular mass indices (LVMI) throughout 3 years of treatment in the pivotal extension trial with MPS II patients aged 5–31 years.5,a

ELAPRASE significantly increased 6MWT distance in the pivotal extension trial with MPS II patients aged 5–31 years, indicating an improvement in physical functioning.1,2,a

The effect of ELAPRASE on 6-minute walking test (6MWT) distance:2

6MWT-layer-01 6MWT-layer-02 6MWT-layer-03
6MWT-layer-04
Adapted by permission from the Copyright Clearance Center: Springer, Genetics in Medicine, Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome, Muenzer et al., 2010.

ELAPRASE significantly reduced liver and spleen size and was shown to efficiently break down glycosaminoglycans (GAGs).​1,2

  • In the pivotal extension trial with MPS II patients aged 5–31 years, ELAPRASE significantly reduced liver and spleen size (p<0.001) from as early as 4 months and was sustained over 3 years.<0.05).1,2,a​

The effect of ELAPRASE on spleen volume:2

liver-volume-layer-01 liver-volume-layer-02 liver-volume-layer-03
liver-volume-layer-04

The effect of ELAPRASE on liver volume​:2

spleen-volume-layer-01 spleen-volume-layer-02 spleen-volume-layer-03
spleen-volume-layer-04
Adapted by permission from the Copyright Clearance Center: Springer, Genetics in Medicine, Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome, Muenzer et al., 2010

ELAPRASE, indicating a reduction in abnormal glycosaminoglycan (GAG) accumulation.1,2

In the pivotal extension trial with MPS II patients aged 5–31 years:a

  • ELAPRASE significantly reduced urinary GAG (uGAG) levels by 4 months and sustained reductions over 3 years.​1

  • ELAPRASE-treated patients typically displayed uGAG levels within the normal range following treatment (67% within normal range
    at 3 years).​1,2

The effect of ELAPRASE on uGAG levels:2

ugag-layer-01 ugag-layer-02 ugag-layer-03
ugag-layer-04
Adapted by permission from the Copyright Clearance Center: Springer, Genetics in Medicine, Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome, Muenzer et al., 2010


ELAPRASE was shown to be effective in treating paediatric patients with MPS II, as was demonstrated by similar safety and efficacy outcomes in a trial of patients aged 16 months to 7.5 years compared to that reported in the pivotal trial.3,4,b

In the trial with MPS II patients aged 16 months to 7.5 years:b

  • ELAPRASE was well tolerated.3,4

    • ELAPRASE was well tolerated in patients aged ≥16 months. All 28 patients experienced >1 AE, all of which were mild to moderate except two cases of sleep apnoea, which did not cause treatment disruption. Most common AEs probably or possibly related to treatment were fever, rash, urticaria, and vomiting.

  • ELAPRASE reduced uGAG output (mean % change -54.4%).​3,4

  • ELAPRASE reduced liver and spleen volumes in young patients by 17.4% and 20.6%, respectively.​4

Mean percentage change from baseline in index of liver size, spleen volume, and urinary GAGs over 53 weeks:4

treating-paediatric-patientslayer-01 treating-paediatric-patientslayer-02 treating-paediatric-patientslayer-03
treating-paediatric-patientslayer-04
Adapted by permission from the Copyright Clearance Center: Springer, Genetics in Medicine, A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4–7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy, Giugliani et al., 2014.


ELAPRASE was shown to be well tolerated in patients with Hunter syndrome.1–4

  • In the pivotal and pivotal extension trial, the total number of adverse events (AEs) was similar in both the placebo and ELAPRASE treatment groups, and the majority were mild to moderate and did not lead to patient discontinuation.1–3

  • In the pivotal study, the number of patients that experienced serious AEs (SAEs) was similar between treatment groups; all except three SAEs were considered unrelated to the study group.​​1

  • The ELAPRASE safety profile in patients aged 16 months to 7.5 years was consistent with that observed in older patients.1–4

  • AEs deemed to be associated with treatment were most commonly infusion-related reactions (IRRs) that were mild to moderate in severity and largely diminished with continued treatment.​1–4


References:

  1. Muenzer J, Wraith J.E, Beck M et al. Genet Med. 2006;8(8):465-473.

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

  3. Elaprase Summary of Product Characteristics. 2020. Accessed February 2021.​

  4. Giugliani R, Hwu W.H., Szymanska A.T. et al. Genet Med. 2014;16(6):435–441.​

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

calculator