ELAPRASE

It is best to tell your doctor or pharmacist about everything, herbs, vitamins, and nutritional supplements before starting treatment.

It must be stored in a refrigerator at a temperature between 2-8 degrees Celsius.

It does not freeze.

In females.

Hunter syndrome (Mucopolysaccharidosis II, MPS II) 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 syndrome, GAG progressively accumulate in the lysosomes of a variety of cells, leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction.

ELAPRASE is intended to provide exogenous enzyme for uptake into cellular lysosomes.

Mannose-6-phosphate (M6P) residues on the oligosaccharide chains allow binding of the enzyme 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. 12.2 Pharmacodynamics Decreases in urinary GAG levels were observed following treatment with ELAPRASE.

The responsiveness of urinary GAG to dosage alterations of ELAPRASE is unknown, and the relationship of urinary GAG to other measures of clinical response has not been established.

Patients who tested positive for anti-idursulfase antibodies (Ab) experienced a less pronounced decrease in urinary GAG levels. 12.3 Pharmacokinetics Clinical Trials in Patients 5 Years and Older The pharmacokinetic characteristics of idursulfase were evaluated in 59 patients with Hunter syndrome.

The serum concentration of idursulfase was quantified using an antigen-specific ELISA assay.

The area under the concentration-time curve (AUC) increased in a greater than dose proportional manner as the dose increased from 0.15 mg/kg to 1.5 mg/kg following a single 1-hour infusion of ELAPRASE.

The pharmacokinetic parameters at the recommended dose regimen (0.5 mg/kg ELAPRASE administered weekly as a 3-hour infusion) were determined at Week and Week in 10 patients 7.7 to 27 years of age (Table 3).

There were no apparent differences in PK parameter values between Week and Week 27 regardless of the antibody status in these patients.

Table 3.

Pharmacokinetic Parameters in

Patients 7.7 to 27 Years of Age Pharmacokinetic Parameter Week 1 Mean (SD) Week 27 Mean (SD) C max (mcg/mL) 1.5 1.1 AUC (min•mcg/mL) 206 169 t 1/2 (min) 44 48 CL (mL/min/kg) 3.0 3.4 V ss (mL/kg) 213 254 Clinical Trial in Patients 7 Years and Younger Idursulfase pharmacokinetics was evaluated in 27 patients with Hunter syndrome 16 months to 7.5 years of age who received ELAPRASE 0.5 mg/kg once weekly as a 3-hour infusion.

The presence of anti-idursulfase antibody (Ab) was associated with a reduced systemic exposure of idursulfase.

Eight of the 18 Ab-positive patients had no measurable idursulfase concentrations.

An additional 9 Ab-positive patients had decreased C max, AUC, and t 1/2 at Week 27 compared to Week 1 (Table 4).

Idursulfase pharmacokinetics was similar between Week and Week in Ab-negative patients (Table 4).

Table 4.

Patients 16 months to 7.5 Years of Age Week 1 Week 27 Pharmacokinetic Parameter (N=27) All Patients Mean (SD) Anti-idursulfase Antibodies (Ab) Positive anti-idursulfase antibody (Ab) is defined as having at least one serum specimen with measurable antibody during study duration. (n=9) Negative Ab Mean (SD) (n=10 Eight of 18 patients with positive Ab had no measurable concentrations at Week 27). Positive Ab Mean (SD) C max (mcg/mL) 1.33 1.40 0.706 AUC (min•mcg/mL) 224 N=26 281 122 N=9 t 1/2 (min) 160 134 84 CL (mL/min/kg) 2.4 2.0 (0. 8) 7.4 V ss (mL/kg) 394 272 829 All patients with the complete gene deletion or large gene rearrangement genotype (n=8) developed Ab at Week 27.

Five of these eight patients had no measurable idursulfase concentrations at Week 27, and three had a lower systemic exposure at Week 27 compared to Week 1.

Idorsulfase is a purified form of

Idorasulfase, a lysosomal enzyme.

Synthesis of recombinant

DNA technology.

Idorsulfase is used to treat

Hunter syndrome, in which large molecules accumulate in cells in the absence

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To learn more information about the side effects resulting from the use of Idorsulfase, which represents the scientific name for Ilabres Click here to learn about the side effects of Ilabres.

It is prohibited to use the medicine without consulting a specialist doctor in the following cases: During pregnancy or while planning a pregnancy.

It should be used with caution under the supervision of a physician in the following cases: Pregnancy or breastfeeding.

The patient should be monitored while receiving doses of Idorsulfase in the following cases: Exposure to complications and symptoms as a result of the infusion, such as skin rash, itching, hives, or high blood pressure.

The medical staff works to reduce the speed of the injection, dilute the solution, or give some And antipyretics, to control symptoms as well.

Patients with serious respiratory diseases, who must be extremely careful when taking antihistamines or other sedatives.

The recommended dosage is 0.5 mg per kg of body weight administered once every week as an intravenous infusion. 2.1 Recommended Dose The recommended dosage regimen of ELAPRASE is 0.5 mg per kg of body weight administered once weekly as an intravenous infusion.

Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. 2.2 Preparation Instructions Prepare and use ELAPRASE according to the following steps using aseptic technique: Determine the total volume of ELAPRASE to be administered and the number of vials needed based on the patient's weight and the recommended dose of 0.5 mg/kg. Patient's weight (kg) × 0.5 mg per kg of ELAPRASE ÷ 2 mg per mL = Total mL of ELAPRASE Total mL of ELAPRASE ÷ 3 mL per vial = Total number of vials Round up to the next whole vial to determine the total number of vials needed.

Remove the required number of vials from the refrigerator to allow them to reach room temperature.

Before withdrawing the

ELAPRASE solution from the vial, visually inspect each vial for particulate matter and discoloration.

ELAPRASE solution should be clear to slightly opalescent and colorless.

Do not use if the solution is discolored or if there is particulate matter in the solution.

Do not shake the

ELAPRASE solution.

Withdraw the calculated volume of

ELAPRASE from the appropriate number of vials.

Add the calculated volume of

ELAPRASE solution to a 100 mL bag of 0.9% Sodium Chloride Injection, USP for intravenous infusion.

Mix gently.

Do not shake the solution. 2.3 Administration Instructions Administer the diluted ELAPRASE solution to patients using a low-protein-binding infusion set equipped with a low-protein-binding 0.2 micrometer (µm) in-line filter.

The total volume of infusion should be administered over a period of 3 hours, which may be gradually reduced to 1 hour if no hypersensitivity reactions are observed.

Patients may require longer infusion times if hypersensitivity reactions occur; however, infusion times should not exceed 8 hours.

The initial infusion rate should be 8 mL per hour for the first 15 minutes.

If the infusion is well tolerated, the rate of infusion may be increased by 8 mL per hour increments every 15 minutes.

The infusion rate should not exceed 100 mL per hour.

The infusion rate may be slowed, temporarily stopped, or discontinued for that visit in the event of hypersensitivity reactions.

ELAPRASE should not be infused with other products in the infusion tubing. 2.4 Storage and Stability ELAPRASE does not contain preservatives; therefore, after dilution with saline, the infusion bags should be used immediately.

If immediate use is not possible, the diluted solution should be stored refrigerated at 2°C to 8°C (36°F to 46 °F) for up to 24 hours.

Other than during infusion, do not store the diluted ELAPRASE solution at room temperature.

Any unused product or waste material should be discarded and disposed of in accordance with local requirements.

is supplied as a sterile injection in a 5 mL Type I glass vial.

The vials are closed with a butyl rubber stopper with fluororesin coating and an aluminum overseal with a blue flip-off plastic cap.

Each carton contains a single vial

NDC 54092-700-01 Store ELAPRASE vials in the carton at 2°C to 8°C (36°F to 46°F) to protect from light.

Do not freeze or shake.

Do not use

ELAPRASE after the expiration date on the vial.

ELAPRASE vials in the carton at 2°C to 8°C (36°F to 46°F) to protect from light.

Do not freeze or shake.

Do not use

ELAPRASE after the expiration date on the vial.

Risk Summary There are no adequate and well-controlled studies with ELAPRASE use in pregnant women.

Available data from a small number of postmarketing cases with ELAPRASE use in pregnancy are insufficient to inform drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes.

In an animal reproduction study, no evidence of adverse effects on pre.

• and post-natal development was observed with twice weekly intravenous administration of idursulfase to pregnant rats from gestation day 6 through lactation day at about 4 times the recommended human weekly dose of 0.5 mg/kg based on body surface area.

The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.

In the

U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

In a pre.

• and post-natal development study, idursulfase was administered to pregnant rats twice weekly, intravenously, from gestation day 6 through lactation day 19.

No significant adverse effects on pre.

• and post-natal development of the offspring were observed at twice weekly intravenous doses up to 12.5 mg/kg (about 4 times the recommended human weekly dose of 0.5 mg/kg based on body surface area).

Clinical trials with

ELAPRASE were conducted in 96 patients with Hunter syndrome, ages to 31 years old, with the majority of the patients in the pediatric (median age 15 years old).

In addition, an open-label, uncontrolled clinical trial was conducted in 28 patients with Hunter syndrome, ages 16 months to 7.5 years old.

Patients 16 months to 5 years of age demonstrated reduction in spleen volume that was similar to that of adults and children 5 years and older.

However, there are no data to support improvement in disease-related symptoms or long term clinical outcome in patients 16 months to 5 years of age.

The safety and effectiveness of

ELAPRASE have not been established in pediatric patients less than 16 months of age.

Clinical studies of

ELAPRASE did not include patients older than 31 years of age.

It is not known whether older patients respond differently from younger patients.