Update: Shire HGT's Investigational Treatment for Type 1 Gaucher Disease
September 25, 2009 - As treatment sites become active and begin to enroll patients for Shire HGT's treatment protocol HGT-GCB-058 (velaglucerase alfa for patients with Type 1 Gaucher disease), information on site
locations will be
available here.
September 8, 2009 - In the United States, the velaglucerase alfa treatment protocol is currently open and enrolling patients.
In order to plan and anticipate needs within the community, we have sent out a letter to Gaucher-treating physicians in
the United States. The letter is available here.
We will continue to keep you updated on our progress via this Web site and through direct communications
with physicians and the Gaucher patient community. If you have questions or feedback, please contact us
at info@GaucherPatients.com.
August 5, 2009 - This web site was launched to communicate that we were aware of the current need for additional enzyme replacement therapy for type 1 Gaucher disease patients. The purpose of this Web site is to share the latest information regarding our product in development for this serious condition.
On July 6, 2009 we announced that at the request of the U.S. Food and Drug Administration (FDA), we filed a treatment protocol for velaglucerase alfa, our investigational enzyme replacement therapy in development for the treatment of type 1 Gaucher disease.
As we announced on August 3, 2009, treatment protocol HGT-GCB-058 was accepted by the FDA. This treatment protocol will allow physicians to provide velaglucerase alfa, in accordance with the conditions of the protocol, in advance of the drug’s final marketing approval and commercial availability in the United States. The drug will initially be offered free of charge under the protocol to facilitate patient access.
We also announced on July 16, 2009 that the FDA has granted us Fast Track status for velaglucerase alfa. This Fast Track designation is intended to expedite the drug review process in instances where the unmet medical need is considered high. It allows us to have an ongoing dialogue with the FDA about our application and also to file sections of our new drug application (NDA) as they become available instead of having to wait to file all the sections at once. It does not reduce the FDA’s official review timeline.
We will keep you updated on our progress via this Web site and through direct communications with physicians and the Gaucher patient community. If you have questions or feedback, please contact us at info@GaucherPatients.com.