LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type I Gaucher disease. Shire is working with the FDA to determine subsequent steps and timing for the filing of its NDA.

Fast Track designation is an FDA approved process that facilitates the development and expedites the review of drugs to treat serious diseases and fill an unmet medical need with the goal of getting important new treatments to patients earlier. This process allows a company to file the sections of the NDA as they become available instead of filing all the sections at once. It also enables the agency to commence its review and proceed on a rolling basis as the additional sections are completed and submitted for review.

Shire is completing a phase III clinical program that includes three phase III controlled studies involving over 100 patients at 24 sites in 10 countries around the world.

On July 6th, Shire announced that it filed a treatment protocol for velaglucerase alfa at the request of the FDA, which if accepted would allow physicians to treat Gaucher disease patients with velaglucerase alfa on an early access basis, ahead of commercial availability in the US. Under the conditions of the treatment protocol, Shire would provide velaglucerase alfa free of charge initially, in order to provide access to patients as quickly as possible.

Velaglucerase alfa is made with Shire's proprietary technology, in a human cell line. The enzyme produced has the exact human amino acid sequence and carries a human glycosylation pattern.

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-divided in three subtypes according to the presence or absence of neurological involvement. It is also the most common genetic disease affecting Ashkenazi Jewish people (Eastern, Central and Northern European ancestry), with a carrier frequency of 1 in 10 (Dr. John Barranger and Dr. Ed Ginns 1989). This panethnic disease involves many organ systems, such as liver, spleen, lungs, brain, metabolism and bone marrow.

Gaucher Disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents.