Myozyme Adverse Reactions

Summary of safety profile: Infantile-onset Pompe disease: In clinical trials, 39 infantile-onset patients were treated with Myozyme for more than three years (168 weeks with a median of 121 weeks; see Pharmacology: Pharmacodynamis under Actions). Most adverse events reported in these 39 patients were due to manifestations of Pompe disease and not related to the administration of Myozyme. Adverse Drug Reactions (ADRs) reported in at least 2 patients are listed in Table 5 by System Organ Class. ADRs were mostly mild to moderate in intensity and almost all occurred during the infusion or during the 2 hours following the infusion (infusion associated reactions, IARs). Serious infusion reactions including urticaria, rales, tachycardia, decreased oxygen saturation, bronchospasm, tachypnea, periorbital edema and hypertension have been reported.
Late-onset Pompe disease: In a placebo-controlled study lasting 78 weeks, 90 patients with late-onset Pompe disease, aged 10 to 70 years, were treated with Myozyme or placebo randomized in a 2:1 ratio (see Pharmacology: Pharmacodynamics under Actions). Overall, the numbers of patients experiencing adverse reactions and serious adverse reactions were comparable between the two groups. The most common adverse reactions observed were IARs. Slightly more patients in the Myozyme group than in the placebo group experienced IARs (28% versus 23%). The majority of these reactions were non-serious, mild to moderate in intensity and resolved spontaneously. Adverse Drug Reactions (ADRs) reported in at least 2 patients are listed in Table 5. Serious adverse reactions reported in 4 patients treated with Myozyme were: Angioedema, chest discomfort, throat tightness, non-cardiac chest pain and supraventricular tachycardia. Reactions in 2 of these patients were IgE-mediated hypersensitivity reactions.
Tabulated list of adverse reactions: See Table 5.

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Description of selected adverse reactions: A small number of patients (<1%) in clinical trials and in the commercial setting developed anaphylactic shock and/or cardiac arrest during Myozyme infusion that required life-support measures.
Reactions generally occurred shortly after initiation of the infusion. Patients presented with a constellation of signs and symptoms, primarily respiratory, cardiovascular, edematous and/or cutaneous in nature (see Precautions).
Recurrent reactions consisting of flu-like illness or a combination of events such as fever, chills, myalgia, arthralgia, pain, or fatigue occurring after completion of infusions and lasting usually for a few days have been observed in some patients treated with alglucosidase alfa. The majority of patients were successfully rechallenged with alglucosidase alfa using lower doses and/or pretreatment with anti-inflammatory drugs and/or corticosteroids and have continued to receive treatment under close clinical supervision.
Patients with moderate to severe or recurrent IARs have been evaluated for alglucosidase alfa specific IgE antibodies; some patients tested positive including some who experienced an anaphylactic reaction.
Nephrotic syndrome as well as severe cutaneous reactions, possibly immune-mediated, have been reported with alglucosidase alfa including ulcerative and necrotizing skin lesions (see Precautions).
Reporting of suspected adverse reactions: Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions.