MTAP Fact Sheet
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical
program to provide access to Myozyme produced by a larger scale manufacturing process
for a limited time until this manufacturing scale is approved by the FDA. Patients
enrolled in MTAP will be provided Myozyme at no cost during the course of the
program. The goals of MTAP are two-fold: (a) to minimize any potential disruption in
the treatment of patients already on therapy; and (b) to ensure that Myozyme is available
for new patients who are in the most urgent clinical need.
General Information
· Beginning immediately, new patients who meet any of the following criteria will
be eligible for enrollment into the Myozyme Temporary Access Program: (1) all
patients between the ages of six and 18, (2) all who require ventilation assistance for
breathing, and (3) all who require a wheelchair for ambulation.
· For new patients who do not meet the MTAP entry criteria, Myozyme treatment
will be delayed until the larger scale manufacturing process is approved for
commercial use.
· Genzyme may invite some patients who are currently on treatment and are over
18 years of age to enroll into MTAP to optimize supply availability. Genzyme will
contact physicians directly regarding the potential transition of any patients currently
on treatment.
· The program will require IRB approval at treatment sites, and like all clinical
programs, patients will be required to sign an informed consent to enroll into MTAP
once their treating site has received IRB approval.
· Genzyme anticipates an FDA decision in the fourth quarter of 2007. Following
the approval of the larger scale manufacturing process, we will help all patients
enrolled in MTAP and new patients waiting to initiate treatment expeditiously
transition to commercial therapy.
· Physicians seeking additional clarification or information should contact
Genzyme Medical Information at 1-800-745-4447, option 2. Patients should contact
Genzyme Treatment Support at 1-800-745-4447, option 3.
MZ/GL/P155A/05/07