Lumizyme

Drug overview for LUMIZYME (alglucosidase alfa):

Generic name: ALGLUCOSIDASE ALFA (AL-gloo-KOE-si-dase AL-fa)
Drug class: Metabolic Disease Enzyme Replacement, Pompe Disease
Therapeutic class: Metabolic Disease Enzyme Replacement Agents

Alglucosidase alfa is a hydrolytic lysosomal glycogen-specific recombinant human acid alpha-glucosidase (GAA), an enzyme that catalyzes the hydrolysis of alpha-1,4- and alpha-1,6-glycosidic linkages of lysosomal glycogen.

No enhanced Uses information available for this drug.

DRUG IMAGES

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The following indications for LUMIZYME (alglucosidase alfa) have been approved by the FDA:

Indications:
Lysosomal alpha-1,4-glucosidase deficiency

Professional Synonyms:
Acid maltase deficiency
Glycogen storage disease type 2
Glycogen storage disease type II
Glycogenosis type II
Pompe's disease

Dosing information for LUMIZYME
DRUG LABEL	DOSING TYPE	DOSING INSTRUCTIONS
LUMIZYME 50 MG VIAL	Maintenance	Adults infuse 20 mg/kg over 4 hour(s) by intravenous route every 2 weeks at a rate of 1 mg/kg/hr for 30 minutes may increase to a max of 7 mg/kg/hr

Moderate List
Cardiopulmonary impairment
Chronic heart failure
Hypervolemia

The following adverse reaction information is available for LUMIZYME (alglucosidase alfa):

Overview
Severe
Less Severe

Adverse reaction overview.

The most common adverse reactions (>=5%) reported with alglucosidase alfa in clinical trials were hypersensitivity reactions (e.g., anaphylaxis, rash, pyrexia, flushing/feeling hot, urticaria, headache, hyperhidrosis, nausea, cough, decreased oxygen saturation, tachycardia, tachypnea, chest discomfort, dizziness, muscle twitching, agitation, cyanosis, erythema, hypertension/increased blood pressure, pallor, rigors, tremor, vomiting, fatigue, myalgia).

There are 32 severe adverse reactions.

More Frequent	Less Frequent
Anaphylaxis Angioedema Bronchospastic pulmonary disease Cyanosis Hypertension Hypoxia Infection Tachypnea Urticaria	Anemia Apnea Bradycardia Hypoventilation Seizure disorder Tachycardia Throat constriction

Rare/Very Rare
Acute respiratory failure Bacterial sepsis Cardiac arrest Cerebrovascular accident Dermal necrosis Dissection of aorta Dyspnea Glomerulonephritis Heart failure Hypersensitivity drug reaction Nephrotic syndrome Pharyngeal edema Pneumonia Pneumothorax Severe dyspnea Ventricular premature beats

There are 44 less severe adverse reactions.

More Frequent	Less Frequent
Agitation Chest pain Cough Diarrhea Dizziness Erythema Fatigue Fever Flushing Headache disorder Hypotension Malaise Myalgia Nausea Periorbital edema Pharyngitis Rhinitis Skin rash Upper abdominal pain Vomiting Wheezing	Back pain Constipation Gastroenteritis Hyperhidrosis Pallor Paresthesia Pruritus of skin Tremor

Rare/Very Rare
Acute abdominal pain Arthralgia Chills Conjunctivitis Drowsy Eye tearing Flu-like symptoms Livedo reticularis Muscle spasm Nervousness Pain Peripheral edema Proteinuria Skin inflammation Syncope

The following precautions are available for LUMIZYME (alglucosidase alfa):

Pediatric
Pregnant
Lactation
Geriatric

Safety and efficacy of alglucosidase alfa have been established in pediatric patients with Pompe disease. Evidence supporting the use of alglucosidase alfa in pediatric patients is derived from an adequate and well-controlled trial in 57 treatment-naive pediatric patients with infantile-onset Pompe disease who were 0.2 months to 3.5 years of age at first infusion and from a randomized, double-blind, placebo-controlled study in patients with late-onset Pompe disease, which included 2 patients who were 16 years of age or younger.

Contraindicated

None

Severe Precaution

None

Management or Monitoring Precaution

None

The available data from postmarketing and published case reports in pregnant women receiving alglucosidase alfa have not identified a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. The decision to use alglucosidase alfa during pregnancy should be individualized. Untreated Pompe disease may result in worsening respiratory and musculoskeletal symptoms in pregnant women.

No adverse embryofetal effects have been observed in animal reproduction studies. A pregnancy exposure registry has been established for alglucosidase alfa to monitor pregnancy outcomes in women exposed to the drug during pregnancy. Pregnant patients and females of reproductive potential should be encouraged to enroll in the registry.

For additional information, visit https://www.registrynxt.com/ or call 1-800-745-4447, extension 15500.

Available data suggest that alglucosidase alfa is distributed into human milk. Adverse effects of alglucosidase alfa on the breastfed infant have not been reported. It is not known whether the drug affects milk production.

To minimize drug exposure to a breastfed infant, the manufacturer states that a lactating woman may consider interrupting breastfeeding, pumping and discarding breast milk during treatment and for 24 hours after administration of alglucosidase alfa. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for alglucosidase alfa and any potential adverse effects on the breastfed child from the drug or from the underlying maternal condition. Lactating women with Pompe disease treated with alglucosidase alfa should be encouraged to enroll in the Pompe disease registry.

Clinical studies of alglucosidase alfa did not include a sufficient number of patients 65 years of age and older to determine whether they respond differently from younger adult patients.

Lysosomal alpha-1,4-glucosidase deficiency
E74.02	Pompe disease