March 19, 2012 Community Update for Pompe Families in the United States who Utilize
Myozyme® (alglucosidase alfa) Produced at the 160 L Manufacturing Scale
We are writing to provide an update to those families who are currently receiving Myozyme (160 L
alglucosidase alfa) in the United States. As you know, in partnership with the patient and health care
provider communities, Genzyme is actively taking steps to enable a long term sustainable solution for
the constrained 160 L supply. As we announced in January, we are proactively enacting a clinical
program, the ADVANCE study, for patients 12 months of age and older using Myozyme in the US. The
objective of this open-label study is to demonstrate efficacy and safety of treatment with alglucosidase
alfa produced at the 4000 L scale in patients 12 months of age and older and previously treated with 160
L scale alglucosidase alfa in the commercial setting.
We want to remind you that beginning on March 30, 2012, Genzyme will cease shipments of
Myozyme 160 L to any US patient 12 months of age and older who is eligible to enroll in the ADVANCE
study; US patients 12 months of age and older who are using Myozyme must be enrolled into the
ADVANCE study to continue receiving alglucosidase alfa therapy after March 30th
Because the ADVANCE study is a clinical trial, the Genzyme team has been communicating directly with
physicians and their health care teams regarding the process of setting up the ADVANCE study at their
sites. There is a significant amount of effort underway at Genzyme by our medical and clinical teams to
partner with each treating physician to expedite the process of getting the ADVANCE study up and
running. We are diligently tracking progress at each treatment site to understand the timeframes for
enrollment into the ADVANCE study for every individual patient and assessing their risk for missed
infusions. While all efforts are being made to minimize the risk for delayed or missed infusions, some
individuals could experience a temporary interruption in therapy depending on their treatment site’s
enrollment status in the ADVANCE study. We are in discussions with some physicians as needed about
possible short-term alternatives, including the potential to travel to an alternate site for a short period
of time, in the event a local site is not approved and available to enroll patients by early April.
We do understand that members of the Pompe community continue to have questions and concerns
about the treatment plan for specific individuals. We encourage anyone with questions to reach out to
their physician or health care facility directly for information about their or their family member’s status
for the ADVANCE study. The US patient associations, the AMDA and the United Pompe Foundation, are
available as a resource for you and Genzyme Patient Advocacy is also available to address questions and
can be reached at 1-800-745-4447, extension 16500. Thank you.