Adverse Event Reporting

It is the responsibility of Genzyme to collect, review, and communicate all relevant information regarding AEs for patients receiving alglucosidase alfa to relevant regulatory authorities in countries where the drug is under clinical investigation or approved for marketing. Therefore, Genzyme strongly encourages physicians to report all AEs to Genzyme Pharmacovigilance.

DEFINITIONS

An AE is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product. The event does not necessarily have to have a causal relationship with alglucosidase alfa treatment.

An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding, for example), symptom or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. This includes any untoward signs or symptoms experienced by the patient from the time of first infusion.

AEs 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 AEs unless they re-occur with the use of the product after the patient has recovered from the pre-existing condition, or represent an exacerbation in intensity or frequency.

An Infusion Associated Reaction (IAR) is any related AE that occurs following onset of the infusion up to and including the post infusion observation period.

Adverse Event Form (pdf)

Serious Adverse Event (SAE)

An SAE is any AE that results in any of the following outcomes:

• Death
• Life-threatening experience
  i.e., the patient was at risk of death at the time of event – does not include events that hypothetically might have caused death if the event were more severe
• Required or prolonged inpatient hospitalization
• Persistent or significant disability/incapacity
  i.e., the event resulted in a substantial disruption of a person’s ability to conduct normal life functions
• Congenital anomaly
• Important medical events that may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed above.

The physician will be asked to assess the severity of the adverse drug/biologic experience 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 type events observed in clinical practice. Severity is not equivalent to seriousness. Below are listed guidelines for severity assessment:

• Mild: Symptom(s) barely noticeable to the subject/patient or does not make the subject/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 subject/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 subject/patient severe discomfort. Severity may cause cessation of treatment with drug/biologic. Treatment for symptom(s) may be given.

Reporting of Adverse Events

All AEs (including SAEs, IARs, and other non-serious AEs) should be reported to Genzyme Pharmacovigilance as soon as possible, even if the event does not appear to be related to the product, preferably within 1 working day of the reporter’s first knowledge of the event. Such communications should be directed to:

United States & Non European Countries

Genzyme Corporation
Pharmacovigilance Department
675 West Kendall Street
Cambridge, MA 02142 USA
Telephone (US): 800-745-4447, Option 2
Outside US: +1-617-768-9000, Option 2
Fax: +1-617-761-8506

Genzyme Europe BV

Pharmacovigilance Department
Gooimeeer 10
1411 DD Naarden
The Netherlands
Telephone: +31-35-699-1299
Fax: +31-35-694-8756

For serious adverse events, a detailed written description that includes copies of relevant patient records, autopsy reports, and other documents as appropriate should be forwarded. AE Reporting Forms are also included in the “Registry Resources” section of the Pompe Registry EDC System. SAEs will be reported by Genzyme to appropriate regulatory agencies in accordance with the relevant regulations and timelines.

Adverse Event Form (pdf)