• SKIP TO CONTENT
  • SKIP NAVIGATION
  • Patient Resources
    • COVID-19 Patient Resource Center
    • Clinical Trial Listings
    • What is Clinical Research?
    • Volunteering for a Clinical Trial
    • Understanding Informed Consent
    • Useful Resources
    • FDA Approved Drugs
  • Professional Resources
    • Research Center Profiles
    • Market Research
    • Benchmark Reports
    • FDA Approved Drugs
    • Training Guides
    • Books
    • eLearning
    • Events
    • Newsletters
    • White Papers
    • SOPs
    • eCFR and Guidances
  • White Papers
  • Clinical Trial Listings
  • Advertise
  • COVID-19
  • Sign In
  • Create Account
  • Sign Out
  • My Account
Home » Directories » FDA Approved Drugs » Elaprase (idursulfase)

AND
  • A
  • B
  • C
  • D
  • E
  • F
  • G
  • H
  • I
  • J
  • K
  • L
  • M
  • N
  • O
  • P
  • Q
  • R
  • S
  • T
  • U
  • V
  • W
  • X
  • Y
  • Z

Elaprase (idursulfase)

  • Profile

Profile

Contact Information

Website: https://www.elaprase.com/

Currently Enrolling Trials

    Show More

    General Information

    Elaprase (idursulfase) is a purified form of the lysosomal enzyme human iduronate-2-sulfatase of recombinant DNA origin. It is designed to replace the natural enzyme, increasing catabolism of certain accumulated glycosaminoglycans (GAG), which abnormally accumulate in multiple tissue types in patients with mucopolysaccharidosis II (MPS-II, or Hunter syndrome).

    Elaprase is specifically indicated for the treatment of Hunter syndrome (Mucopolysaccharidosis II, MPS II) in adults and children ages 5 and older. The drug has been shown to improve walking capacity in these subjects.

    Elaprase is supplied as sterile, aqueous clear to slightly-opalescent colorless solution for intravenous infusion. Recommended initial dosing is 0.5 mg/kg administered via weekly infusion. The initial recommended infusion rate is 8 ml/hr for the first 15 minutes, which may be increased at 8/ml/hr increments at 15 minute intervals barring appearance of infusion reactions, to a maximum of 100 ml/hr. Total infusion duration is generally 1-3 hours, and should not exceed 8 hours. Infusions may be slowed, temporarily stopped, or discontinued for that dose if infusion reactions occur (see Side Effects, below).

    Side Effects

    Adverse events associated with the use of Elaprase may include, but are not limited to, the following:

    • pyrexia
    • headache
    • arthralgia
    • limb pain
    • pruitis
    • hypertension
    • malaise
    • visual disturbance

    In addition, Elaprase was shown to cause anaphylactic reactions in some patients during infusions. It is contraindicated in patients with comprised respiratory function or acute respiratory disease.

    Mechanism of Action

    Hunter's Syndrome is an X-linked recessive disease caused by insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. This enzyme cleaves the terminal 2-O-sulfate moieties from the glycosaminoglycans (GAG) dermatan sulfate and heparan sulfate. Due to the missing or defective iduronate-2-sulfatase enzyme in patients with Hunter's Syndrome, GAG progressively accumulate in the lysosomes of a variety of cells, leading to cellular engorgement, organomegaly, tissue destruction and organ system dysfunction. Treatment of Hunter's Syndrome patients with Elaprase provides exogenous enzyme for uptake into cellular lysosomes. Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow specific binding of the enzymes to the M6P receptors on the cell surface, leading to cellular internalization of the enzyme, targeting to intracellular lysosomes and subsequent catabolism of accumulated GAG.

    Clinical Trial Results

    FDA approval of Elaprase was based on the results of one clinical trial. This randomized, double-blind, placebo controlled trial enrolled 96 subjects with Hunter's Syndrom, aged 5-31 years. Subjects received Elaprase 0.5mg/kg every week, Elaprase 0.5mg/kg every other week or placebo. The treatment duration was 53 weeks. The primary efficacy endpoint was a score based on the change from baseline to week 53 in distance walked during a 6 minute walk test and the change in forced vital capacity percentages. The primary endpoints showed the greatest statistical significant difference between the weekly Elaprase treated group and the placebo group (p=0.0049). The subjects in the Elaprase weekly group showed a 35 meter mean increase in distance walked versus the placebo group. There was not a statistically significant difference in forced vital capacity percentages between the groups.

    Approval Date: 2006-07-01
    Company Name: Takeda Pharmaceuticals
    Back to Listings

    Upcoming Events

    • 07Jun

      Developing World-Class SOPs: Optimizing Quality and Compliance

    • 08Jun

      Implementing ICH E8 R1 Recommendations Increases Site and Participant Relationship Scoring Measures

    Featured Products

    • Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

      Spreadsheet Validation: Tools and Techniques to Make Data in Excel Compliant

    • Surviving an FDA GCP Inspection

      Surviving an FDA GCP Inspection: Resources for Investigators, Sponsors, CROs and IRBs

    Featured Stories

    • MAGI East 2023

      MAGI East 2023 Preview: Janssen Reports on Environmental Impact of Trials

    • Complexity-360x240.png

      Phase 3 Trials Significantly Rising in Complexity, Says CSDD

    • Quality Level Scale

      Build Quality into Trials Like You’d Build a House, Says FDA’s BIMO Director

    • DE&I

      Trust-Building, Community Connection Still Essential to DE&I Efforts, Experts Say

    Standard Operating Procedures for Risk-Based Monitoring of Clinical Trials

    The information you need to adapt your monitoring plan to changing times.

    Learn More Here
    • About Us
    • Contact Us
    • Privacy Policy
    • Do Not Sell or Share My Data

    Footer Logo

    300 N. Washington St., Suite 200, Falls Church, VA 22046, USA

    Phone 703.538.7600 – Toll free 888.838.5578

    Copyright © 2023. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing