Alglucosidase alfa

Alglucosidase alfa
Black Box Warning
Adult Indications & Dosage
Pediatric Indications & Dosage
Contraindications
Warnings & Precautions
Adverse Reactions
Drug Interactions
Use in Specific Populations
Administration & Monitoring
Overdosage
Pharmacology
Clinical Studies
How Supplied
Images
Patient Counseling Information
Precautions with Alcohol
Brand Names
Look-Alike Names

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Sree Teja Yelamanchili, MBBS [2]

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Black Box Warning

WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE See full prescribing information for complete Boxed Warning. * Life-threatening anaphylactic reactions and severe hypersensitivity reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria, have occurred in some patients during and after alglucosidase alfa infusions. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Closely observe patients during and after alglucosidase alfa administration and be prepared to manage anaphylaxis and hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and have them seek immediate medical care should signs and symptoms occur. Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload, and require additional monitoring.

Overview

Alglucosidase alfa is an enzyme replacement therapy that is FDA approved for the treatment of patients with Pompe disease (GAA deficiency-Glycogen storage disease-type II). There is a Black Box Warning for this drug as shown here. Common adverse reactions include {{{adverseReactions}}}.

Adult Indications and Dosage

FDA-Labeled Indications and Dosage (Adult)

There is limited information regarding Alglucosidase alfa FDA-Labeled Indications and Dosage (Adult) in the drug label.

Off-Label Use and Dosage (Adult)

Guideline-Supported Use

There is limited information regarding Off-Label Guideline-Supported Use of Alglucosidase alfa in adult patients.

Non–Guideline-Supported Use

There is limited information regarding Off-Label Non–Guideline-Supported Use of Alglucosidase alfa in adult patients.

Pediatric Indications and Dosage

FDA-Labeled Indications and Dosage (Pediatric)

Indications

Hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase (GAA) deficiency)

Dosage

Recommended Dose

• The recommended dosage of alglucosidase alfa is 20 mg/kg body weight administered every 2 weeks as an intravenous infusion.
• For injection: 50 mg of alglucosidase alfa as lyophilized powder in a single-use vial for reconstitution

Instructions for Use

• Alglucosidase alfa does not contain any preservatives. Vials are single-use only. Discard any unused product.
• The total volume of infusion is determined by the patient's body weight and should be administered over approximately 4 hours.
• Infusions should be administered in a step-wise manner using an infusion pump.
• The initial infusion rate should be no more than 1 mg/kg/hr.
• The infusion rate may be increased by 2 mg/kg/hr every 30 minutes, after patient tolerance to the infusion rate is established, until a maximum rate of 7 mg/kg/hr is reached.
• Vital signs should be obtained at the end of each step.
• If the patient is stable, alglucosidase alfa may be administered at the maximum rate of 7 mg/kg/hr until the infusion is completed.
• The infusion rate may be slowed or temporarily stopped in the event of mild to moderate hypersensitivity reactions.
• In the event of anaphylaxis or severe hypersensitivity reaction, immediately discontinue administration of alglucosidase alfa, and initiate appropriate medical treatment.
• See TABLE below for the rate of infusion at each step, expressed as mL/hr based on the recommended infusion volume by patient weight.

Reconstitution, Dilution, and Administration

• Alglucosidase alfa should be reconstituted, diluted and administered by a healthcare professional.
• Use aseptic technique during preparation. Do not use filter needles during preparation.
• Determine the number of vials to be reconstituted based on the individual patient's weight and the recommended dose of 20 mg/kg.
• Patient weight (kg) × dose (mg/kg) = patient dose (in mg)
• Patient dose (in mg) divided by 50 mg/vial = number of vials to reconstitute. If the number of vials includes a fraction, round up to the next whole number.

• Example: Patient weight (68 kg) × dose (20 mg/kg) = patient dose (1,360 mg)

 1,360 mg divided by 50 mg/vial = 27.2 vials; therefore, 28 vials should be reconstituted.

• Remove the required number of vials from the refrigerator and allow them to reach room temperature prior to reconstitution (approximately 30 minutes).
• Reconstitute each alglucosidase alfa vial by slowly injecting 10.3 mL of sterile water for Injection, USP to the inside wall of each vial. Each vial will yield a concentration of 5 mg/mL.
• The total extractable dose per vial is 50 mg per 10 mL.
• Avoid forceful impact of the water for injection on the powder and avoid foaming.
• This is done by slow drop-wise addition of the water for injection down the inside of the vial and not directly onto the lyophilized cake.
• Tilt and roll each vial gently. Do not invert, swirl, or shake.
• The reconstituted alglucosidase alfa solution should be protected from light.
• Perform an immediate visual inspection on the reconstituted vials for particulate matter and discoloration.
• If upon immediate inspection opaque particles are observed or if the solution is discolored do not use.
• The reconstituted solution may occasionally contain some alglucosidase alfa particles (typically less than 10 in a vial) in the form of thin white strands or translucent fibers subsequent to the initial inspection. This may also happen following dilution for infusion. These particles have been shown to contain alglucosidase alfa and may appear after the initial reconstitution step and increase over time. Studies have shown that these particles are removed via in-line filtration without having a detectable effect on the purity or strength.
• Alglucosidase alfa should be diluted in 0.9% Sodium Chloride for Injection, USP, immediately after reconstitution, to a final alglucosidase alfa concentration of 0.5 to 4 mg/mL.
• Slowly withdraw the reconstituted solution from each vial. Avoid foaming in the syringe.
• Remove airspace from the infusion bag to minimize particle formation due to the sensitivity of alglucosidase alfa to air-liquid interfaces.
• Add the reconstituted alglucosidase alfa solution slowly and directly into the sodium chloride solution. Do not add directly into airspace that may remain within the infusion bag. Avoid foaming in the infusion bag.
• Gently invert or massage the infusion bag to mix. Do not shake.
• Administer alglucosidase alfa using an in-line low protein binding 0.2 µm filter.
• Do not infuse alglucosidase alfa in the same intravenous line with other products.
• The reconstituted and diluted solution should be administered without delay. If immediate use is not possible, the reconstituted and diluted solution is stable for up to 24 hours at 2°C to 8°C (36°F to 46°F).
• Storage of the reconstituted solution at room temperature is not recommended.
• The reconstituted and diluted alglucosidase alfa solution should be protected from light.
• Do not freeze or shake.
• Alglucosidase alfa does not contain any preservatives. Vials are single-use only.
• Discard any unused product.

Dosage form and Strengths

For injection: 50 mg of alglucosidase alfa is supplied as a sterile, nonpyrogenic, white to off-white, lyophilized cake or powder in a single-use vial for reconstitution. After reconstitution, the resultant solution concentration is 5 mg/mL.

Off-Label Use and Dosage (Pediatric)

Guideline-Supported Use

There is limited information regarding Off-Label Guideline-Supported Use of Alglucosidase alfa in pediatric patients.

Non–Guideline-Supported Use

There is limited information regarding Off-Label Non–Guideline-Supported Use of Alglucosidase alfa in pediatric patients.

Contraindications

There is limited information regarding Alglucosidase alfa Contraindications in the drug label.

Warnings

WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE See full prescribing information for complete Boxed Warning. * Life-threatening anaphylactic reactions and severe hypersensitivity reactions, presenting as respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria, have occurred in some patients during and after alglucosidase alfa infusions. Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following alglucosidase alfa treatment. Closely observe patients during and after alglucosidase alfa administration and be prepared to manage anaphylaxis and hypersensitivity reactions. Inform patients of the signs and symptoms of anaphylaxis, hypersensitivity reactions, and immune-mediated reactions and have them seek immediate medical care should signs and symptoms occur. Infantile-onset Pompe disease patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload, and require additional monitoring.

There is limited information regarding Alglucosidase alfa Warnings' in the drug label.

Adverse Reactions

Clinical Trials Experience

There is limited information regarding Alglucosidase alfa Clinical Trials Experience in the drug label.

Postmarketing Experience

There is limited information regarding Alglucosidase alfa Postmarketing Experience in the drug label.

Drug Interactions

There is limited information regarding Alglucosidase alfa Drug Interactions in the drug label.

Use in Specific Populations

Pregnancy

Pregnancy Category (FDA): There is no FDA guidance on usage of Alglucosidase alfa in women who are pregnant.
Pregnancy Category (AUS): There is no Australian Drug Evaluation Committee (ADEC) guidance on usage of Alglucosidase alfa in women who are pregnant.

Labor and Delivery

There is no FDA guidance on use of Alglucosidase alfa during labor and delivery.

Nursing Mothers

There is no FDA guidance on the use of Alglucosidase alfa in women who are nursing.

Pediatric Use

There is no FDA guidance on the use of Alglucosidase alfa in pediatric settings.

Geriatic Use

There is no FDA guidance on the use of Alglucosidase alfa in geriatric settings.

Gender

There is no FDA guidance on the use of Alglucosidase alfa with respect to specific gender populations.

Race

There is no FDA guidance on the use of Alglucosidase alfa with respect to specific racial populations.

Renal Impairment

There is no FDA guidance on the use of Alglucosidase alfa in patients with renal impairment.

Hepatic Impairment

There is no FDA guidance on the use of Alglucosidase alfa in patients with hepatic impairment.

Females of Reproductive Potential and Males

There is no FDA guidance on the use of Alglucosidase alfa in women of reproductive potentials and males.

Immunocompromised Patients

There is no FDA guidance one the use of Alglucosidase alfa in patients who are immunocompromised.

Administration and Monitoring

Administration

There is limited information regarding Alglucosidase alfa Administration in the drug label.

Monitoring

There is limited information regarding Alglucosidase alfa Monitoring in the drug label.

IV Compatibility

There is limited information regarding the compatibility of Alglucosidase alfa and IV administrations.

Overdosage

There is limited information regarding Alglucosidase alfa overdosage. If you suspect drug poisoning or overdose, please contact the National Poison Help hotline (1-800-222-1222) immediately.

Pharmacology

There is limited information regarding Alglucosidase alfa Pharmacology in the drug label.

Mechanism of Action

There is limited information regarding Alglucosidase alfa Mechanism of Action in the drug label.

Structure

There is limited information regarding Alglucosidase alfa Structure in the drug label.

Pharmacodynamics

There is limited information regarding Alglucosidase alfa Pharmacodynamics in the drug label.

Pharmacokinetics

There is limited information regarding Alglucosidase alfa Pharmacokinetics in the drug label.

Nonclinical Toxicology

There is limited information regarding Alglucosidase alfa Nonclinical Toxicology in the drug label.

Clinical Studies

There is limited information regarding Alglucosidase alfa Clinical Studies in the drug label.

How Supplied

There is limited information regarding Alglucosidase alfa How Supplied in the drug label.

Storage

There is limited information regarding Alglucosidase alfa Storage in the drug label.

Images

Drug Images

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Package and Label Display Panel

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Patient Counseling Information

There is limited information regarding Alglucosidase alfa Patient Counseling Information in the drug label.

Precautions with Alcohol

Alcohol-Alglucosidase alfa interaction has not been established. Talk to your doctor about the effects of taking alcohol with this medication.

Brand Names

There is limited information regarding Alglucosidase alfa Brand Names in the drug label.

Look-Alike Drug Names

There is limited information regarding Alglucosidase alfa Look-Alike Drug Names in the drug label.

Drug Shortage Status

Price

References

The contents of this FDA label are provided by the National Library of Medicine.

Alglucosidase alfa

Clinical data
ATC code	A16AB07 (WHO)
Identifiers
CAS Number	[1] 420794-05-0
E number	{{#property:P628}}
ECHA InfoCard	{{#property:P2566}}
Chemical and physical data
Formula	C4758H7262N1274O1369S35
Molar mass	105338

Alglucosidase alfa (Myozyme) is a drug used to treat Glycogen storage disease type II (Pompe's disease). It is an analogue of acid alpha-glucosidase.

Some health plans have refused to pay for Myozyme, because it costs $300,000 a year for adults, and has only been approved by the F.D.A. for use in infants.