Important Information about MYOZYME® (alglucosidase alfa)
MYOZYME (alglucosidase alfa) is indicated for use in patients with Pompe disease (GAA deficiency). MYOZYME has been shown to improve ventilator-free survival in patients with infantile-onset Pompe disease as compared to an untreated historical control, whereas use of MYOZYME in patients with other forms of Pompe disease has not been adequately studied to assure safety and efficacy.
WARNING
Risk of Anaphylaxis
Life-threatening anaphylactic reactions have been observed in some patients during MYOZYME infusions. Therefore, appropriate medical support should be readily available when MYOZYME is administered.
Risk of Cardiorespiratory Failure
Patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to infusion reactions, and require additional monitoring.
Risk of Anaphylaxis and Allergic Reactions: Life-threatening and severe allergic reactions have included anaphylactic shock, cardiac arrest, respiratory distress, hypotension, bradycardia, hypoxia, bronchospasm, throat tightness, dyspnea, angioedema, and urticaria. In clinical trials and postmarketing safety experience with MYOZYME, approximately 1% of patients developed anaphylactic shock and/or cardiac arrest during MYOZYME infusion that required life-support measures. In clinical trials and expanded access programs with MYOZYME, approximately 14% of patients treated with MYOZYME have developed allergic reactions that involved at least 2 of 3 body systems, cutaneous, respiratory, or cardiovascular systems. (Please see WARNINGS section of the Full Prescribing Information.)
Risk of Acute Cardiorespiratory Failure: Acute cardiorespiratory failure requiring intubation and inotropic support has been observed up to 72 hours after infusion with MYOZYME in infantile-onset Pompe disease patients with underlying cardiac hypertrophy, possibly associated with fluid overload with intravenous administration of MYOZYME. (Please see Full Prescribing Information for appropriate infusion volumes.)
Risk of Cardiac Arrhythmia and Sudden Cardiac Death During General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter in infantile-onset Pompe disease patients with cardiac hypertrophy.
Infusion Reactions: The most common adverse reactions requiring intervention were infusion-related reactions which occurred in 20 of 39 (51%) of patients treated with MYOZYME in clinical studies. Some reactions were severe. Severe infusion reactions reported in more than 1 patient in clinical studies and the expanded access program included: fever, decreased oxygen saturation, tachycardia, cyanosis, and hypotension. Other infusion reactions reported in more than one patient in clinical studies and the expanded access program included: rash, flushing, urticaria, fever, cough, tachycardia, decreased oxygen saturation, vomiting, tachypnea, agitation, increased blood pressure/hypertension, cyanosis, irritability, pallor, pruritus, retching, rigors, tremor, hypotension, bronchospasm, erythema, face edema, feeling hot, headache, hyperhidrosis, increased lacrimation, livedo reticularis, nausea, periorbital edema, restlessness, and wheezing.
Some patients were pre-treated with antihistamines, antipyretics and/or steroids. Infusion reactions occurred in some patients after receiving antipyretics, antihistamines, or steroids. Infusion reactions may occur at any time during, or up to 2 hours after, the infusion of MYOZYME, and are more likely with higher infusion rates.
In addition to the infusion reactions reported in clinical trials and expanded access programs, the following have been reported during postmarketing use of MYOZYME: cardiac arrest, pharyngeal edema, peripheral edema, chest pain, chest discomfort, muscle spasm, fatigue and conjunctivitis.
Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from infusion reactions. Therefore, these patients should be monitored more closely during administration of MYOZYME. Patients with an acute underlying illness at the time of MYOZYME infusion appear to be at greater risk for infusion reactions. Careful consideration should be given to the patient’s clinical status prior to administration of MYOZYME.
Immune Mediated Reactions: Severe cutaneous and systemic immune mediated reactions have been reported in postmarketing experience with MYOZYME in at least 2 patients. Patients should be monitored for the development of systemic immune complex-mediated reactions involving skin and other organs while receiving MYOZYME.
Immunogenicity: The majority of patients in clinical trials developed antibodies to treatment with MYOZYME. The effect of antibody development on the long term efficacy of MYOZYME is not fully understood. There is an observation that some patients, who develop high and sustained anti-alglucosidase alfa antibody titers, including those who possess 2 null mutations, have a poorer clinical response.
MYOZYME is available by prescription only.
Adverse reactions should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. To learn more, please see the Full Prescribing Information or contact Genzyme Medical Information at 1-800-745-4447, option 2.
