ELAPRASE
ELAPRASE is a hydrolytic lysosomal glycosaminoglycan-specific enzyme indicated for the treatment of patients with Hunter syndrome, also known as Mucopolysaccharidosis II (MPS II). The therapy is approved for use in adults and pediatric patients 16 months of age and older. In patients five years and older, the drug is used to improve walking capacity, while in patients aged 16 months to five years, it has been shown to reduce spleen volume.
How ELAPRASE Works
Hunter syndrome is characterized by insufficient levels of iduronate-2-sulfatase, an enzyme required to break down glycosaminoglycans (GAG) that otherwise accumulate and cause organ system dysfunction. ELAPRASE provides an exogenous replacement enzyme that binds to mannose-6-phosphate receptors on the cell surface, allowing it to be internalized and targeted to intracellular lysosomes. Once inside the lysosomes, the enzyme catabolizes accumulated GAG by cleaving specific sulfate moieties, thereby reducing cellular engorgement and tissue destruction.
Details
- Status
- Prescription
- First Approved
- 2006-07-24
- Routes
- IV (INFUSION)
- Dosage Forms
- INJECTABLE
ELAPRASE Approval History
What ELAPRASE Treats
2 indicationsELAPRASE is approved for 2 conditions since its original approval in 2006. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hunter Syndrome
- Mucopolysaccharidosis II
ELAPRASE Boxed Warning
RISK OF ANAPHYLAXIS Life-threatening anaphylactic reactions have occurred in some patients during and up to 24 hours after ELAPRASE infusions. Anaphylaxis, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have been reported to occur during and after ELAPRASE infusions, regardless of duration of the course of treatment. Closely observe patients during and after ELAPRASE administration and be prepared to manage anaphylaxis. Inform patients o...
WARNING: RISK OF ANAPHYLAXIS Life-threatening anaphylactic reactions have occurred in some patients during and up to 24 hours after ELAPRASE infusions. Anaphylaxis, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have been reported to occur during and after ELAPRASE infusions, regardless of duration of the course of treatment. Closely observe patients during and after ELAPRASE administration and be prepared to manage anaphylaxis. Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions and require additional monitoring [see Warnings and Precautions (5.1 , 5.3) and Adverse Reactions (6) ] . WARNING: RISK OF ANAPHYLAXIS See full prescribing information for complete boxed warning. Life-threatening anaphylactic reactions, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have occurred in some patients during and up to 24 hours after ELAPRASE infusions. Closely observe patients during and after ELAPRASE administration and be prepared to manage anaphylaxis. Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions and require additional monitoring. ( 5.1 , 5.3 , 6 )
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ELAPRASE FDA Label Details
ProIndications & Usage
FDA Label (PDF)ELAPRASE is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). ELAPRASE has been shown to improve walking capacity in patients 5 years and older. In patients 16 months to 5 years of age, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment with ELAPRASE has reduced spleen volume similarly to that of adults and children 5 years of age and older. The safety and efficacy of ELAPRASE have not been established in pediatric patients less than 16 months of age [see Use in Specific Populations ]. ELA...
WARNING: RISK OF ANAPHYLAXIS Life-threatening anaphylactic reactions have occurred in some patients during and up to 24 hours after ELAPRASE infusions. Anaphylaxis, presenting as respiratory distress, hypoxia, hypotension, urticaria and/or angioedema of throat or tongue have been reported to occur d...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.