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Myozyme is a brand name for Alglucosidase Alfa. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency). Myozyme is indicated in adults and paediatric patients of all ages.
Verbatim from this product's EMA label. Tap a section to expand.
Myozyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases. Posology The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body weight administered once every 2 weeks.
Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease. Paediatric and older people There is no evidence for special considerations when Myozyme is administered to paediatric patients of all ages or older people.
Patients with renal and hepatic impairment The safety and efficacy of Myozyme in patients with renal or hepatic impairment have not been evaluated and no specific dose regimen can be recommended for these patients. 3 Method of administration Myozyme should be administered as an intravenous infusion.
Infusions should be administered incrementally. It is recommended that the infusion begin at an initial rate of 1 mg/kg/h and be gradually increased by 2 mg/kg/h every 30 minutes if there are no signs of infusion associated reactions (IARs) until a maximum rate of 7 mg/kg/h is reached.
8. Home infusion Infusion of Myozyme at home may be considered for patients who are tolerating their infusions well and have no history of moderate or severe IARs for a few months. The decision to have a patient move to home infusion should be made after evaluation and upon recommendation by the treating physician.
Home infusion infrastructure, resources, and procedures, including training, must be established and available to the healthcare professional. Home infusion should be supervised by a healthcare professional who should be always available during the home infusion and for a specified time after infusion.
Dose and infusion rate should remain constant while at home, and not be changed without supervision of a healthcare professional. Appropriate information should be given by the treating physician and/or nurse to the patient and/or caregiver prior to initiation of home infusion.
4). Subsequent infusions may need to occur in a hospital or in an appropriate setting of outpatient care until no such adverse reaction is present. 6.
8. Home infusion Infusion of Myozyme at home may be considered for patients who are tolerating their infusions well and have no history of moderate or severe IARs for a few months. The decision to have a patient move to home infusion should be made after evaluation and upon recommendation by the treating physician.
Home infusion infrastructure, resources, and procedures, including training, must be established and available to the healthcare professional. Home infusion should be supervised by a healthcare professional who should be always available during the home infusion and for a specified time after infusion.
Dose and infusion rate should remain constant while at home, and not be changed without supervision of a healthcare professional. Appropriate information should be given by the treating physician and/or nurse to the patient and/or caregiver prior to initiation of home infusion.
4). Subsequent infusions may need to occur in a hospital or in an appropriate setting of outpatient care until no such adverse reaction is present. 6. 8). 4 Special warnings and precautions for use Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.
8). Because of the potential for severe infusion associated reactions, appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available when Myozyme is administered. If severe hypersensitivity or anaphylactic reactions occur, immediate discontinuation of Myozyme infusion should be considered and appropriate medical treatment should be initiated.
The current medical standards for emergency treatment of anaphylactic reactions are to be observed. Infusion Associated Reactions Approximately half of the patients treated with Myozyme in infantile-onset clinical studies and 28% of the patients treated with Myozyme in a late-onset clinical study developed infusion associated reactions (IARs).
Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded. 8). Because of the potential for severe infusion associated reactions, appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available when Myozyme is administered.
If severe hypersensitivity or anaphylactic reactions occur, immediate discontinuation of Myozyme infusion should be considered and appropriate medical treatment should be initiated. The current medical standards for emergency treatment of anaphylactic reactions are to be observed.
Infusion Associated Reactions Approximately half of the patients treated with Myozyme in infantile-onset clinical studies and 28% of the patients treated with Myozyme in a late-onset clinical study developed infusion associated reactions (IARs).
IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion. 8). A tendency was 4 observed in infantile patients treated with a higher dose (40 mg/kg) to experience more symptoms when developing IARs.
Infantile onset patients who develop high IgG antibody titres appear to be at higher risk for developing more frequent IARs. However, IARs occurred regardless of antibody titres. g. pneumonia, sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs.
Careful consideration should be given to the patient’s clinical status prior to administration of Myozyme. Patients should be closely monitored and all cases of IARs, delayed reactions and possible immunological reactions should be reported to the marketing authorisation holder.
8). Mild and transient effects may not require medical treatment or discontinuation of the infusion. Reduction of the infusion rate, temporary interruption of the infusion, or pre-treatment, generally with oral antihistamine and/or antipyretics and/or corticosteroids, has effectively managed most reactions.
8).
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IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion. 8). A tendency was 4 observed in infantile patients treated with a higher dose (40 mg/kg) to experience more symptoms when developing IARs.
Infantile onset patients who develop high IgG antibody titres appear to be at higher risk for developing more frequent IARs. However, IARs occurred regardless of antibody titres. g. pneumonia, sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs.
Careful consideration should be given to the patient’s clinical status prior to administration of Myozyme. Patients should be closely monitored and all cases of IARs, delayed reactions and possible immunological reactions should be reported to the marketing authorisation holder.
8). Mild and transient effects may not require medical treatment or discontinuation of the infusion. Reduction of the infusion rate, temporary interruption of the infusion, or pre-treatment, generally with oral antihistamine and/or antipyretics and/or corticosteroids, has effectively managed most reactions.
IARs may occur at any time during the infusion of Myozyme or generally up to 2 hours after, and are more likely with higher infusion rates. 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 associated reactions.
Therefore, these patients should be monitored more closely during administration of Myozyme. Immunogenicity The effect of IgG antibody formation on safety and efficacy has been evaluated in clinical trials and post-marketing experience.
In clinical studies, the majority of patients developed IgG antibodies to alglucosidase alfa and seroconversion typically occured within 3 months of treatment. Thus, development of IgG antibodies is expected to occur in most patients treated with Myozyme.
Overall, a correlation was not observed between the onset of IARs and the time of IgG antibody formation. IARs can occur across all levels of antibody titres, however a trend was observed for more frequent IARs with higher titres of IgG antibody.
The clinical impact on efficacy is multifactorial, however the development of high and sustained IgG antibody titres is a contributing factor. With regard to IOPD, a tendency was observed for patients treated with a higher dose (40 mg/kg) to develop higher titres of IgG antibodies.
Furthermore, Cross Reactive Immunologic Material (CRIM) status has been shown to be associated with immunogenicity and patients’ responses to enzyme replacement therapies. Negative CRIM status, indicating no endogenous enzyme is detected, is a risk factor to develop high and sustained IgG antibody titres.
This risk is higher among CRIM negative patients versus CRIM-positive patients and is a contributing factor to a poor outcome . However, high and sustained IgG antibody titres has also occurred in a limited number of […]
IARs may occur at any time during the infusion of Myozyme or generally up to 2 hours after, and are more likely with higher infusion rates. 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 associated reactions.
Therefore, these patients should be monitored more closely during administration of Myozyme. Immunogenicity The effect of IgG antibody formation on safety and efficacy has been evaluated in clinical trials and post-marketing experience.
In clinical studies, the majority of patients developed IgG antibodies to alglucosidase alfa and seroconversion typically occured within 3 months of treatment. Thus, development of IgG antibodies is expected to occur in most patients treated with Myozyme.
Overall, a correlation was not observed between the onset of IARs and the time of IgG antibody formation. IARs can occur across all levels of antibody titres, however a trend was observed for more frequent IARs with higher titres of IgG antibody.
The clinical impact on efficacy is multifactorial, however the development of high and sustained IgG antibody titres is a contributing factor. With regard to IOPD, a tendency was observed for patients treated with a higher dose (40 mg/kg) to develop higher titres of IgG antibodies.
Furthermore, Cross Reactive Immunologic Material (CRIM) status has been shown to be associated with immunogenicity and patients’ responses to enzyme replacement therapies. Negative CRIM status, indicating no endogenous enzyme is detected, is a risk factor to develop high and sustained IgG antibody titres.
This risk is higher among CRIM negative patients versus CRIM-positive patients and is a contributing factor to a poor outcome . However, high and sustained IgG antibody titres has also occurred in a limited number of CRIM-positive patients, generally with very low endogenous enzyme.
With respect to LOPD patients, the majority showed either stabilizing or decreasing antibody titres over time. The development of high and sustained IgG antibody titres is infrequent in LOPD patients. Thus, the impact of IgG antibodies is more limited for LOPD patients.
IgG antibody titres should be monitored based on clinical phenotype. Baseline serum sample collection prior to the first infusion is strongly encouraged. For IOPD patients, regular monitoring during first year of treatment (example: every 3 months) is suggested and subsequent monitoring depending on clinical outcomes and antibody titres level.
For LOPD patients, antibody development should be assessed within 6 months and subsequent monitoring as clinically warranted based on safety and efficacy considerations. Patients who experience hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis.
8). Therefore, these patients should be monitored more closely during 5 administration of Myozyme. Some IgE positive patients were successfully rechallenged with Myozyme using a slower infusion rate at lower initial doses and have continued to receive Myozyme under close clinical supervision.
Immune-mediated reactions Severe cutaneous reactions, possibly immune mediated, have been reported with alglucosidase alfa, including ulcerative and necrotizing skin […]