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alglucosidase alfa (Rx)Brand and Other Names:Lumizyme, Myozyme

 
 
 

Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

powder for injectable solution

  • 50mg/vial (Lumizyme)
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Pompe Disease (GAA Deficiency)

20 mg/kg IV q2wk; infuse over ~4 hr 

Infusion rate: 1 mg/kg/hr IV initially, may increase by 2 mg/kg/hr q30min, not to exceed 7 mg/kg/hr

Dosage Forms & Strengths

powder for injectable solution

  • 5mg/vial (Myozyme)
  • 50mg/vial (Lumizyme)
more...

Pompe Disease (GAA Deficiency)

20 mg/kg IV q2wk; infuse over ~4 hr 

Infusion rate: 1 mg/kg/hr IV initially, may increase by 2 mg/kg/hr q30min, not to exceed 7 mg/kg/hr

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Adverse Effects

>10%

Pyrexia (92%)

Diarrhea (62%)

Rash (54%)

Infusion reactions (51%)

Vomiting (49%)

Cough (46%)

Diaper dermatitis (46%)

Pneumonia (46%)

Otitis media (44%)

URI (44%)

O2 saturation decreased (41%)

Gastroenteritis (41%)

Pharyngitis (36%)

Respiratory distress (33%)

Respiratory failure (31%)

Anemia (31%)

Oral candidiasis (31%)

Rhinorrhea (28%)

Catheter-related infection (28%)

GERD (26%)

Post-procedural pain (26%)

Bronchiolitis (23%)

Constipation (23%)

Nasopharyngitis (23%)

Tachycardia (23%)

Tachypnea (23%)

Bradycardia (21%)

Flushing (21%)

Urticaria (21%)

Frequency Not Defined

Bronchospasm

Blurred vision

Abdominal pain

Agitation

Dyspnea

Facial Erythema

Insomnia

Irritability

Pallor

Restlessness

Tremor

Vertigo

Coronary artery disease

Hypokalemia

Conjunctivitis

Postmarketing Reports

Anaphylactic shock, respiratory failure, respiratory arrest, cardiac arrest, hypoxia, dyspnea, wheezing, convulsions, peripheral coldness, restlessness, nervousness, back pain, stridor, pharyngeal edema, abdominal pain, apnea, muscle spasm, conjunctivitis, hyperparathyroidism

Proteinuria and nephrotic syndrome secondary to membranous glomerulonephritis, and necrotizing skin lesions reported

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Warnings

Black Box Warnings

Anaphylaxis and severe hypersensitivity

  • Life-threatening anaphylactic reactions and severe hypersensitivity reactions reported after IV infusions
  • Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions reported
  • Closely observe patients during and after administration and be prepared to manage anaphylaxis and hypersensitivity reactions
  • Inform patients and caregivers 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

Compromised cardiac or respiratory function

  • 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
  • Additional is monitoring required in these patients

Contraindications

None

Cautions

Anaphylaxis and hypersensitivity reactions have been observed in patients during and up to 3 hr after infusion; some of the reactions were life-threatening and included anaphylactic shock, cardiac arrest, respiratory arrest, respiratory distress, hypoxia, apnea, dyspnea, bradycardia, tachycardia, bronchospasm, throat tightness, hypotension, angioedema (including tongue or lip swelling, periorbital edema, and face edema), and urticaria Immune-mediated cutaneous reactions have been reported including necrotizing skin lesions; these reactions occurred several weeks to 3 yr after initiation

Severe reactions are managed with infusion interruption, administration of antihistamines, corticosteroids, intravenous fluids, and/or oxygen, when clinically indicated; epinephrine may be administered; appropriate medical support, including cardiopulmonary resuscitation equipment, should be readily available when alglucosidase alfa is administered

Consider risks and benefits of re-administering alglucosidase alfa following anaphylactic or hypersensitivity reaction; monitor closely with appropriate resuscitation measures available, if decision is made to re-administer product

Nephrotic syndrome secondary to membranous glomerulonephritis was observed in some Pompe disease patients treated with alglucosidase alfa who had persistently positive anti-rhGAA IgG antibody titers; patients receiving alglucosidase alfa should undergo periodic urinalysis

Patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise during infusions

General anesthesia can be complicated by the presence of severe cardiac and skeletal (including respiratory) muscle weakness; extreme caution should be used when administering general anesthesia; ventricular arrhythmias and bradycardia, resulting in cardiac arrest or death, or requiring cardiac resuscitation or defibrillation have been observed in patients with infantile-onset Pompe disease with cardiac hypertrophy during general anesthesia for central venous catheter placement

As with all therapeutic proteins, there is potential for immunogenicity; monitor patients for IgG antibody formation every 3 months for 2 years and annually thereafter; consider testing for IgG titers if patients develop hypersensitivity reactions, other immune-mediated reactions, or lose clinical response; may also test patients who experience reduced clinical response for inhibitory antibody activity; patients who experience anaphylactic or hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis

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Pregnancy & Lactation

Pregnancy Category: C

Lactation: Enzymatic activity of alglucosidase alfa detected in breast milk of a lactating woman up to 24 hr after end of intravenous alglucosidase alfa administration; may temporarily pump and discard breast milk produced during 24 hr after administration of alglucosidase alfa to minimize infant exposure to alglucosidase alfa; exercise caution when administering alglucosidase alfa to nursing mother

Pregnancy Categories

A:Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

B:May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

C:Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

D:Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

X:Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

NA:Information not available.

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Pharmacology

Mechanism of Action

Recombinant acid alpha-glucosidase (GAA)

Carbohydrate groups on GAA molecule bind to mannose-6-phosphate receptors, then GAA is transported into cell where it undergoes proteolytic cleavage resulting in increased enzymatic glycogen cleavage

Pharmacokinetics

Half-Life: 2-3 hr

Peak Plasma Concentration: 162±31 mcg/m

AUC: 811±141 mcg-hr/mL

Vd: 80-110 mL/kg

Clearance: 20-30 mL/hr/kg

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Administration

IV Incompatibilities

Do not mix with other drugs in same infusion line

IV Preparation

Do not use filter needles during preparation

Determine number of vials required for dose (20 mg/kg)

Remove the required number of vials from the refrigerator and allow them to reach room temperature prior to reconstitution (approximately 30 minutes)

Reconstitute each vial by slowly injecting 10.3 mL sterile water for injection

Avoid foaming & forceful addition of fluid

Protect reconstituted solution from light

Discard if discoloration or particulate matter present

Dilute (slowly) reconstituted solution in 0.9% NaCl to final concentration of 0.5-4 mg/mL

Filter diluted solution through 0.2 micron low protein-binding, in-line filter during administration

IV Administration

Administer by IV infusion over ~4 hr

1 mg/kg/hr IV infusion initially, may increase by 2 mg/kg/hr q30 min; not to exceed 7 mg/kg/hr

Recommended infusion volume:

  • 1.25-10 kg: 50 mL
  • 10.1-20 kg: 100 mL
  • 20.1-30 kg: 150 mL
  • 30.1-35 kg: 200 mL
  • 35.1-50 kg: 250 mL
  • 50.1-60 kg: 300 mL
  • 60.1-100 kg: 500 mL
  • 100.1-120 kg: 600 mL
  • 120.1-140 kg: 700 mL
  • 140.1-160 kg: 800 mL
  • 160.1-180 kg: 900 mL
  • 180.1-200 kg: 1000 mL

Storage

Unreconstituted vials: Store at 2-8°C (36-46°F)

Protect from freezing

Do not shake

Reconstituted/diluted solution stable up to 24 hr at 2-8°C (36-46°F)

Protect from light

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Images

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Formulary

FormularyPatient Discounts

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The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

Tier Description
1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
NC NOT COVERED – Drugs that are not covered by the plan.
Code Definition
PA Prior Authorization
Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
QL Quantity Limits
Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
ST Step Therapy
Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
OR Other Restrictions
Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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