Fabrazyme Adverse Event Reporting

Safety Information for patients receiving Fabrazyme^® (agalsidase beta)
The most serious and most common adverse reactions reported with Fabrazyme^® are infusion reactions. Infusion reactions may include tachycardia, hypertension, throat tightness, chest pain/tightness, dyspnea, fever, chills/rigors, abdominal pain, pruritus, urticaria, nausea, vomiting, lip or ear edema, rash, hypotension, myalgia, and headache. Patients may be given antipyretics prior to infusion. Serious infusion reactions may occur after extended durations of Fabrazyme treatment. Patients with compromised cardiac function who receive Fabrazyme should be monitored closely. Physicians should consider testing for IgE in patients who experienced suspected allergic reactions and consider the risks and benefits of continued treatment in patients who test positive to IgE. The safety and effectiveness of Fabrazyme in pediatric patients have not been established.

Side effects should be reported promptly to Genzyme. Fabrazyme is available by prescription only. For more information, please see full prescribing information.

Adverse Event Reporting
It is the responsibility of Genzyme to collect, review, and communicate all relevant information regarding adverse events (AEs) for patients receiving Fabrazyme (agalsidase beta) to the regulatory authorities in countries where the drug is in clinical investigation or is approved for marketing.

Definitions

An AE is any undesirable physical, psychological or behavioral effect experienced by a patient, in conjunction with the use of the drug or biologic, whether or not product-related. This includes any untoward signs or symptoms experienced by the patient or subject from the time of first infusion.

Adverse events may include, but are not limited to:

• Subjective or objective symptoms spontaneously offered by the patient or subject and/or observed by the physician or medical staff.
• Laboratory abnormalities of clinical significance.

Disease signs, symptoms and/or laboratory abnormalities already existing prior to the use of the product are not considered adverse experiences unless they re-occur after the patient has recovered from the pre-existing condition, or represent an exacerbation in intensity or frequency.

Adverse events considered serious, as defined below, must be reported on the AE Report Form and faxed to Genzyme within 24 hours.

Adverse Event Form – US & International (PDF)
Adverse Event Form – Europe (PDF)

A serious adverse event involves an occurrence that at any dose:

• Results in death
• Is life-threatening

Any event in which the patient was at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe.

• Requires inpatient hospitalization or prolongation of an existing hospitalization
• Results in persistent or significant disability/incapacity

The adverse event that resulted in a substantial disruption of a person’s ability to conduct normal life functions.

• Is a congenital anomaly/birth defect
• Is an important medical event

Any event that, based upon appropriate medical judgment, may jeopardize the patient and may require medical or surgical intervention to prevent one of the outcomes listed above.

The physician will be asked to provide the severity of the AE using the following categories: mild, moderate and severe. This assessment is subjective and the physician should use medical judgment to compare the reported AE to similar types of events observed in clinical practice. It is important to recognize that severity is not equivalent to event seriousness. Guidelines for severity assessments are listed below:

• Mild: Symptom(s) barely noticeable to the patient or does not make the patient uncomfortable. The AE does not influence performance or functioning. Prescription drugs are not ordinarily needed for relief of symptom(s).
• Moderate: Symptom(s) of a sufficient severity to make the patient uncomfortable. Performance of daily activities is influenced. Treatment of symptom(s) may be needed.
• Severe: Symptom(s) of a sufficient severity to cause the patient severe discomfort. Severity may cause cessation of treatment with the drug. Treatment for symptom(s) may be given.

Reporting of Adverse Events

All adverse events should be reported to Genzyme, even if the event does not appear to be related to the product. Such communications should be directed to:

United States & Non-European Countries Europe
Genzyme Corporation
Medical Affairs
Cambridge, Massachusetts USA
Telephone: within the USA: 800-745-4447, option 2
outside the USA: +617-252-7500, option 2
FAX: +617-761-8506

Genzyme Europe BV
Pharmacovigilance Department
The Netherlands
Telephone: +31-35-699-1431
FAX: +31-35-694-8756

Serious adverse event reports should be reported to the appropriate Genzyme Medical Affairs/Pharmacovigilance department within 24 hours. A detailed written description that includes copies of relevant patient records, autopsy reports and other documents as appropriate should also be forwarded. SAEs will be reported by Genzyme to appropriate Regulatory Agencies in accordance with the timetable and relevant regulations.

Non-serious adverse events should also be reported to the appropriate Genzyme Pharmacovigilance department in a timely manner by faxing a completed AE Event Form.

Adverse Event Form – US & International (PDF)
Adverse Event Form – Europe (PDF)