JOINT LETTER FROM THE AMDA AND UPF

Dear All,

The AMDA, UPF, NORD, and Genzyme attended a stakeholder’s meeting on August 29, 2007 at the FDA to discuss the current Myozyme supply situation. Following is a brief report on the meeting.

Representatives from Genzyme included individuals from the executive, regulatory, and patient advocacy departments.  The director of the Division of Gastroenterology Products of the FDA headed the meeting, and other FDA representatives included the Inborn Errors of Metabolism Division, the Drug Shortage Division, and the Patient Liaison Division. All parties expressed their understanding of the seriousness of the situation, and their dedication to doing what was best for the patients.

The FDA is in the process of reviewing data on Myozyme produced from a 2000-liter production platform. Although this platform was approved by the EMEA (European Regulatory Authority) and is currently in use in 33 countries throughout the world, the FDA’s drug approval process is independent. It is not linked to the approval by other regulatory agencies.

Genzyme revealed that Myozyme from both production methods (the approved 160-liter and the unapproved 2000-liter) led to the clearing of glycogen from muscle cells in mouse models, and that information derived from the personal experiences of doctors and patients in Europe indicates that the Myozyme from the larger platform is safe and effective. However, it was reiterated to all parties that clinical data was necessary before the FDA could grant approval of this production platform. The FDA made it clear that it was their duty to safeguard drug approval and use in the United States.

After presentations by both the FDA and Genzyme, the patient groups were given the opportunity to ask questions. During the ensuing discussion, we had the chance to explain the concerns of the patient population, and the difficulties some are facing in enrolling in MTAP. By the conclusion of the meeting, we felt that all parties understood that there are no easy answers in this situation. MTAP was a first step to alleviate the supply problems, but there are new programs being discussed by the FDA and Genzyme in order to offer access to Myozyme from the larger production platform to those in a “critical condition.” We will bring you further information as soon as it becomes available.

If you have any questions or concerns, please feel free to contact us.

Thank you,


Tiffany House                                                            David Hamlin

AMDA                                                                         UPF

utsatiff@aol.com                                                      DHAMLIN02@aol.com