Alglucosidase Alfa

Alglucosidase Alfa is a prescription medication used for the treatment of Pompe Disease (GAA Deficiency).

• Alglucosidase Alfa is available under the following different brand names: Lumizyme, Myozyme

Common side effects of Alglucosidase Alfa include:

• Hives,
• Difficulty breathing,
• Swelling of the face, lips, tongue, or throat,
• Pale skin,
• Blue lips,
• Feeling hot or feverish,
• Skin rash,
• Itching,
• Flushing (warmth, redness, or tingly feeling),
• Headache,
• Dizziness,
• Agitation,
• Muscle twitching,
• Fast heart rate,
• Rapid breathing,
• Chest discomfort,
• Cough,
• Increased blood pressure,
• Nausea,
• Vomiting,
• Tremor,
• Shaking,
• Increased sweating,
• Muscle pain, and
• Tiredness

Serious side effects of Alglucosidase Alfa include:

• Hives,
• Difficulty breathing,
• Swelling of the face, lips, tongue, or throat,
• Lightheadedness,
• Slow heart rate,
• Weak pulse,
• Slow breathing or breathing that may stop,
• Wheezing,
• Tight feeling in the chest or throat,
• Pale skin,
• Cold or clammy skin,
• Blue lips or fingernails,
• Cold hands or feet,
• Warmth, redness, numbness, or tingly feeling under the skin,
• Rash,
• Itching,
• Sweating,
• Fever,
• Severe headache,
• Nausea,
• Restlessness,
• Nervousness,
• Seizure,
• Fast heart rate,
• Pounding in the neck or ears,
• Anxiety,
• Confusion,
• Skin changes (sores, lesions, ulcers, discoloring),
• Puffy eyes,
• Swelling of your ankles or feet,
• Weight gain, and
• Urine that looks foamy

Rare side effects of Alglucosidase Alfa include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Powder for injectable solution

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

Adult and pediatric dosage

• 20 mg/kg intravenous every 2 weeks; infuse over approximately 4 hours.  
• Infusion rate: 1 mg/kg/hour intravenous initially, may increase by 2 mg/kg/hour every 30 mins, not to exceed 7 mg/kg/hour.

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Alglucosidase Alfa has no noted severe interactions with any other drugs.
• Alglucosidase Alfa has no noted serious interactions with any other drugs.
• Alglucosidase Alfa has no noted moderate interactions with any other drugs.
• Alglucosidase Alfa has no noted minor interactions with any other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, or if you have health questions or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Alglucosidase Alfa?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Alglucosidase Alfa?”

Cautions

• Anaphylaxis and hypersensitivity reactions have been observed in patients during and up to 3 hours 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 years after initiation
• Severe reactions are managed with infusion interruption, and 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 the risks and benefits of re-administering Alglucosidase Alfa following an anaphylactic or hypersensitivity reaction; monitor closely with appropriate resuscitation measures available if the decision is made to re-administer the 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.
• Immune-Mediated Reactions
  • Immune tolerance induction administered in conjunction with may also aid tolerability of Alglucosidase Alfa under the management of a clinical specialist knowledgeable in immune tolerance induction in pediatric Pompe disease to optimize treatment.
  • Immune tolerance induction administered before and in conjunction with initiation of Alglucosidase Alfa reported to aid tolerability of Alglucosidase Alfa in CRIM-negative patients; CRIM status is associated with immunogenicity and patients’ responses to enzyme replacement therapies.
  • CRIM-negative infants with infantile-onset Pompe disease treated with Alglucosidase Alfa have shown poorer clinical response in the presence of high sustained IgG antibody titers and positive inhibitory antibodies compared to CRIM-positive infants.

Pregnancy and Lactation

• Data from postmarketing reports and published case reports with Alglucosidase Alfa use in pregnant women have not identified an associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes; the continuation of treatment during pregnancy should be individualized to the pregnant woman; untreated disease may result in worsening disease symptoms in pregnant women.
• Pregnant women and women of reproductive potential should be encouraged to enroll in the Pompe patient registry; the registry will monitor the effect of therapy on pregnant women and their offspring; for more information, visit www.registrynxt.com or call 1-800-745-4447, extension 15500.
• Disease-associated maternal and/or embryo-fetal risk; untreated Pompe disease has been associated with worsening respiratory and musculoskeletal symptoms in some pregnant women.
• Lactation
  • Available published literature suggests the presence of Alglucosidase Alfa in human milk; there are no reports of adverse effects on the breastfed infant; there is no information on the effects of Alglucosidase Alfa on milk production; developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for therapy and any potential adverse effects on the breastfed child from the drug or from the underlying maternal condition.
  • Lactating women with Pompe disease receiving treatment should be encouraged to enroll in the Pompe disease registry.
  • A lactating woman may consider interrupting breastfeeding, pumping, and discarding breast milk during treatment and for 24 hours after therapy administration to minimize drug exposure to a breastfed infant.