Shire Reports Positive Results From First of Three Phase III Trials of velaglucerase alfa for Type 1 Gaucher Disease and Provides Important Updates on Interaction...(and resolved withoutsequelae. Th...),Health

"These findings are very encouraging. They illustrate important potential benefits that velaglucerase alfa may provide to patients who are affected by Type 1 Gaucher disease," said Dr. Atul Mehta, Clinical Director of the Lysosomal Storage Disorders Unit, Royal Free Hospital, London. "Velaglucerase alfa appears to be an excellent choice for Type 1 Gaucher patients. The prospect of having another treatment option available to help patients achieve therapeutic goals is very important and is welcomed by both the physicians and patients."

Regulatory Updates

With regard to ongoing interactions with the FDA, Shire provided the following important updates:

    - The FDA has accepted Shire's treatment protocol for velaglucerase alfa.
    The acceptance of the treatment protocol by the FDA will enable
    physicians to treat Gaucher patients with velaglucerase alfa prior to
    commercialization. Shire will initially provide velaglucerase alfa free
    of charge to patients who are enrolled in the protocol.

    - Shire has begun a rolling submission of a New Drug Application (NDA) to
    the FDA for velaglucerase alfa to treat patients with Type 1 Gaucher
    disease. The submission was initiated on July 30, 2009, three weeks
    after Shire received Fast Track designation. Fast Track designation
    allows a company to file the sections of the NDA as they become available
    and enables the agency to commence its review on a rolling basis. The
    company expects to complete the NDA submission by the end of this
    quarter.

Background on Gaucher disease

Gaucher disease is an autosomal recessive disease and the most prevalent Lysosomal Storage Disorder (LSD), with an incidence of about 1 in 20,000 live births. Despite the fact that Gaucher Disease consists of a phenotype, with varying degrees of severity, it has been sub-divide
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