ELAPRASE

Elaprase is an intravenous infusion that is utilized for the treatment of certain symptoms associated with Hunter’s syndrome, also known as mucopolysaccharidosis (MYOO-koe-pol-ee-SAK-a-rye-DOE-sis). This condition is caused by a genetic disorder that results in the deficiency of a natural enzyme. Elaprase aids in the replacement of this enzyme that is lacking in some individuals.

Hunter syndrome results from a mutation in the IDS gene, responsible for producing the I2S enzyme. This enzyme is crucial for breaking down glycosaminoglycans (GAGs) in the lysosomes of cells.

Hunter syndrome is a rare genetic disorder characterized by the inability of the body to effectively break down specific sugar molecules. This leads to the accumulation of these molecules in organs and tissues, resulting in damage that can impact both physical and mental development.

Approval Date: July 24, 2006. Please note that documents in PDF format will require Adobe Acrobat Reader® for viewing.

ELAPRASE is a medication that contains idursulfase, a purified version of the human I2S enzyme. This enzyme is lacking or faulty in individuals with Hunter syndrome. ELAPRASE is created to mimic the natural enzyme and is manufactured using recombinant DNA technology in a human cell line.

The mechanism of action involves the replacement of the iduronate-2-sulfatase enzyme, which is responsible for the hydrolysis of mucopolysaccharides such as dermatan sulfate and heparan sulfate in different cells.

The contents of each vial of ELAPRASE include 6 mg of idursulfase, 24 mg of sodium chloride, 6.75 mg of sodium phosphate monobasic monohydrate, 2.97 mg of sodium phosphate dibasic heptahydrate, and 0.66 mg of polysorbate 20. It is important to note that ELAPRASE does not contain any preservatives. Each vial is intended for single use only.

Mucopolysaccharidoses are a collection of genetic metabolic disorders that occur when the body lacks or has faulty enzymes required to break down glycosaminoglycans, which are complex sugar molecules found in our cells.

ELAPRASE is a concentrated solution that needs to be diluted in 100 mL of 0.9% Sodium Chloride for Injection before intravenous infusion. Each vial of ELAPRASE contains 3 mL (6 mg) of idursulfase and should only be used once. It is recommended to use an infusion set with a 0.2 micron (µm) filter.

ELAPRASE has been approved by the FDA since 2006 and is currently available in 77 countries.

There is no available data to show that ELAPRASE improves walking ability in patients aged 16 months to 5 years. However, it has been shown to improve walking capacity in patients aged 5 years and older.

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The price of a 3 milliliter supply of Elaprase intravenous solution (2 mg/mL) can vary, but it is typically around $3,312 at different pharmacies.

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During the studies, Elaprase demonstrated the benefit of improving lung function and enhancing the walking ability of the patients. Initially, the patients were able to walk an average distance of approximately 395 meters in six minutes.

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Elaprase (idursulfase) is a drug that has the potential to enhance mobility and reduce spleen enlargement in individuals diagnosed with Hunter syndrome.

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ELAPRASE is a type of treatment known as enzyme replacement therapy (ERT). It contains a specific enzyme called iduronate-2-sulfatase (I2S), which is lacking or insufficient in individuals with Hunter syndrome. ELAPRASE is created through recombinant DNA technology in a human cell line, resulting in a purified form of the I2S enzyme.

Idursulfase is classified as an enzyme replacement therapy (ERT).

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Physicians may consider home infusions as an appropriate option for patients who tolerate ELAPRASE infusions well. However, it is important that these home infusions are conducted under the supervision of a physician or other healthcare professionals, following approved labeling guidelines.

Medicare provides coverage for the majority of injectable and infused drugs when administered by a licensed medical professional, as these drugs are typically not self-administered.

ELAPRASE is given through an IV and comes in a clear to slightly opalescent, colorless solution. Before it is given, it needs to be diluted with 0.9% Sodium Chloride Injection, USP. The solution is sterile and does not contain any pyrogens.

IV therapy has revolutionized the treatment of various chronic illnesses, including Crohn’s disease, ulcerative colitis (UC), multiple sclerosis (MS), rheumatoid arthritis (RA), migraines, and autoimmune disorders. These conditions can now be effectively managed with long-term relief through the use of advanced pharmaceuticals administered intravenously.

Elaprase (idursulfase) is a drug that has the potential to enhance mobility and reduce spleen enlargement in individuals diagnosed with Hunter syndrome.

IV therapy is frequently employed in hospitals when patients are unable to take medications orally or when an intravenous route is deemed more efficient. It is utilized for various purposes such as treating severe infections, cancer, dehydration, gastrointestinal disorders, and autoimmune diseases.

Some examples of medications include blood factors, corticosteroids, erythropoietin, infliximab, inotropic heart medications, growth hormones, immunoglobulin, natalizumab, and various others.

Elaprase contains idursulfase, which is a synthetic version of the human enzyme iduronate-2-sulfatase. This enzyme is used to replace the deficient or defective enzyme in individuals with Hunter syndrome.

Elaprase was designated as an ‘orphan medicine’ on 11 December 2001 due to the low number of patients with Hunter syndrome, classifying it as a rare disease.