As you may be aware, in response to the almost year-long Fabrazyme supply shortage and at the request of the FDA, Shire developed two early access programs for REPLAGAL® (agalsidase alfa) – our investigational enzyme replacement therapy for Fabry disease – to support the U.S. patient community. Shire established a U.S. treatment protocol for REPLAGAL and we are also supporting emergency INDs.
Recently, Fabry patients and their physicians have shown rapidly increasing interest in joining the U.S. treatment protocol. Due to this high demand combined with the uncertainty inherent in any regulatory submission, Shire decided to close enrollment in the treatment protocol as of 3pm ET on Friday, June 25th.
We have provided additional information about this decision for physicians and patients on their pages of this website.
If you have questions, please call Medical Information at 1-866-888-0660 or submit them online via the "Ask Shire" tool on this website.
Get Updates from Shire
As REPLAGAL® is not approved in the U.S., Shire cannot contact Fabry patients directly unless they have consented to receive communications from the company. If you are interested in receiving updates from Shire please sign up here.
I'd like to receive updates from Shire »