ELELYSO

ELELYSO- taliglucerase alfa injection, powder, lyophilized, for solution
Pfizer Laboratories Div Pfizer Inc

1 INDICATIONS AND USAGE

ELELYSO is indicated for the treatment of patients 4 years and older with a confirmed diagnosis of Type 1 Gaucher disease.

2 DOSAGE AND ADMINISTRATION

2.1 Recommended Dosage in Patients 4 Years and Older

Treatment-naïve patients: The recommended dosage of ELELYSO for long-term treatment is 60 units/kg of body weight administered every other week as a 60 to 120 minute intravenous infusion.

Patients switching from imiglucerase: Patients currently being treated with imiglucerase for Type 1 Gaucher disease can be switched to ELELYSO. Patients previously treated on a stable dosage of imiglucerase are recommended to begin treatment with ELELYSO at that same units/kg dosage when they switch from imiglucerase to ELELYSO. Administer ELELYSO for long-term treatment every other week as a 60 to 120 minute intravenous infusion. Dosage adjustments can be made based on achievement and maintenance of each patient’s therapeutic goals [see Clinical Studies (14.2)].

2.2 Preparation Instructions

ELELYSO should be reconstituted, diluted, and administered under the supervision of a healthcare professional.

Each vial of ELELYSO provides 200 units of taliglucerase alfa and is intended for one-time use and for only one patient. The reconstitution and dilution steps must be completed using aseptic technique.

ELELYSO should be reconstituted with Sterile Water for Injection, USP and diluted with 0.9% Sodium Chloride Injection, USP, to a final volume of 100 mL to 200 mL, and administered by intravenous infusion.

Prepare ELELYSO according to the following steps. Use aseptic technique.

a.
Determine the number of vials to be reconstituted based on the patient’s weight and the recommended dose of 60 units/kg, using the following calculations (1–3):
(1)
Total dose in units = Patient’s weight (kg) × dose (units/kg)
(2)
Total number of vials = Total dose in units divided by 200 units/vial
(3)
Round up to the next whole vial.
b.
Remove the required number of vials from the refrigerator. Do not leave these vials at room temperature longer than 24 hours prior to reconstitution. Do not heat or microwave these vials.
c.
Reconstitute each vial of ELELYSO with 5.1 mL of Sterile Water for Injection, USP to yield a reconstituted product with a concentration of 40 units/mL and an extractable volume of 5 mL. Upon reconstitution, mix vials gently. DO NOT SHAKE. Prior to further dilution, visually inspect the solution in the vials; the solution should be clear and colorless. Do not use if the solution is discolored or if foreign particulate matter is present.
d.
Withdraw the calculated dose of drug from the appropriate number of vials and dilute with 0.9% Sodium Chloride Injection, USP, to a final volume of 100 to 200 mL.
i.
For pediatric patients, a final volume of 100 to 120 mL should be used.
ii.
For adult patients, a final volume of 130 to 150 mL may be used. However, if the volume of reconstituted product alone is equal to or greater than 130 to 150 mL, then the final volume should not exceed 200 mL.
e.
Mix gently. DO NOT SHAKE. Since this is a protein solution, slight flocculation (described as translucent fibers) occurs occasionally after dilution.

2.3 Administration Instructions

After reconstitution and dilution, the preparation should be administered via intravenous infusion and filtered through an in-line low protein-binding 0.2 μm filter.

  • For pediatric patients: An initial infusion rate of 1 mL/minute should be used. After tolerability to ELELYSO is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2 mL/minute. The total volume of the infusion should be delivered over a minimum of 60 minutes.
  • For adult patients: An initial infusion rate of 1.2 mL/minute should be used. After tolerability to ELELYSO is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2.2 mL/minute. The total volume of the infusion should be delivered over a minimum of 60 minutes.

As ELELYSO contains no preservative, the product should be used immediately once reconstituted. If immediate use is not possible, the reconstituted product may be stored for up to 24 hours refrigerated at 2 °C to 8° C (36 °F to 46 °F) under protection from light or up to 4 hours at room temperature (20 °C to 25 °C (68 °F to 77 °F) without protection from light. The diluted product may be stored for up to 24 hours at 2 °C to 8 °C (36 °F to 46 °F) under protection from light. Storage of the reconstituted product and the diluted product should not exceed a total of 24 hours. Do not freeze. Discard any unused product.

3 DOSAGE FORMS AND STRENGTHS

For injection: 200 units white to off-white lyophilized powder in a single-dose vial for reconstitution.

4 CONTRAINDICATIONS

None.

5 WARNINGS AND PRECAUTIONS

5.1 Hypersensitivity Reactions Including Anaphylaxis

Serious hypersensitivity reactions, including anaphylaxis, have occurred in some patients treated with ELELYSO. In clinical trials, 2 of 72 (3%) patients treated with ELELYSO experienced signs and symptoms consistent with anaphylaxis. Signs and symptoms of these patients included urticaria, hypotension, flushing, wheezing, chest tightness, nausea, vomiting, and dizziness. These reactions occurred during ELELYSO infusion.

In clinical trials with ELELYSO, 21 of 72 (29%) patients experienced hypersensitivity reactions, including anaphylaxis. Signs and symptoms of hypersensitivity reactions included pruritus, angioedema, flushing, erythema, rash, nausea, vomiting, cough, chest tightness, and throat irritation. These reactions have occurred up to 3 hours after the start of infusion [see Adverse Reactions (6.1)].

Due to the potential for anaphylaxis, appropriate medical support should be readily available when ELELYSO is administered. Observe patients closely for an appropriate period of time after administration of ELELYSO, taking into account the time to onset of anaphylaxis seen in clinical trials. Inform patients of the signs and symptoms of anaphylaxis, and instruct them to seek immediate medical care should signs and symptoms occur. If anaphylaxis occurs, discontinue ELELYSO immediately, and initiate appropriate medical treatment.

Management of hypersensitivity reactions should be based on the severity of the reaction and includes slowing or temporary interruption of the infusion and/or administration of antihistamines, antipyretics, and/or corticosteroids for mild reactions. Pretreatment with antihistamines and/or corticosteroids may prevent subsequent hypersensitivity reactions. Patients were not routinely premedicated prior to infusion of ELELYSO during clinical studies. If severe hypersensitivity reactions occur, immediately stop the infusion of ELELYSO and initiate appropriate treatment.

Consider the risks and benefits of re-administering ELELYSO in patients who have experienced a severe reaction associated with ELELYSO. Caution should be exercised upon rechallenge, and appropriate medical support should be readily available [see Adverse Reactions (6.3)].

6 ADVERSE REACTIONS

6.1 Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

Clinical Trials of ELELYSO as Initial Therapy

  • Clinical Trial in Patients 19 Years and Older

The safety of ELELYSO at dosages of either 30 units/kg (n=16) or 60 units/kg (n=16) every other week was assessed in 32 adult treatment-naïve patients (aged 19 to 74 years) with Type 1 Gaucher disease in a 9-month double-blind, randomized clinical trial.

Table 1: Adverse Reactions in ≥5% of Treatment-Naïve Adult Patients Treated with ELELYSO
Preferred Term Treatment-Naïve Adults (N=32)n (%)
Headache 6 (19)
Arthralgia 4 (13)
Fatigue 3 (9)
Nausea 3 (9)
Dizziness 3 (9)
Abdominal pain 2 (6)
Pruritus 2 (6)
Flushing 2 (6)
Vomiting 2 (6)
Urticaria 2 (6)
  • Clinical Trial in Patients 16 Years and Younger

    The safety of ELELYSO at dosages of either 30 units/kg (n=4) or 60 units/kg (n=5) every other week was assessed in 9 pediatric treatment-naïve patients (aged 2 to 13 years) with Type 1 Gaucher disease in a 12-month randomized clinical trial.

    The most common adverse reaction (≥10%) was vomiting, which occurred in 4 of 9 patients. Two patients developed hypersensitivity reactions; one patient experienced severe vomiting and gastrointestinal inflammation, and 1 experienced mild throat irritation and chest discomfort. Both patients responded to treatment with antihistamines and continued ELELYSO treatment.

Clinical Trial in Patients Switching from Imiglucerase Treatment to ELELYSO

The safety of ELELYSO was assessed in 31 patients (26 adult and 5 pediatric patients), ages 6 to 66 years old, with Type 1 Gaucher disease who had previously been receiving treatment with imiglucerase for a minimum of 2 years. ELELYSO was administered for 9 months at the same number of units as each patient’s previous imiglucerase dose.

Table 2: Adverse Reactions in ≥10% of Patients Switched from Imiglucerase to ELELYSO (after 9 months on treatment)
Preferred Term Patients Switched from Imiglucerase(N=31; 26 adults and 5 children)n (%)
Arthralgia 4 (13)
Headache 4 (13)
Pain in extremity 3 (10)
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