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MTAP Fact Sheet MTAP, the Myozyme®…

Tags: 18 years, alfa, ambulation, clarification, disruption, entry criteria, fact sheet, fda, fda decision, fourth quarter, genzyme, informed consent, limited time, manufacturing process, mtap, myozyme, physicians, transition, ventilation, wheelchair,
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Language: english
Created: Thu May 3 14:59:47 2007
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                                   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