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Shire plc, the globalspecialty biopharmaceutical company, announces it has received FastTrack designation from the US Food and Drug Administration (FDA) forvelaglucerase alfa, its enzyme replacement therapy in development forthe treatment of Type I Gaucher disease. Shire is working with the FDAto determine subsequent steps and timing for the filing of its NDA.
FastTrack designation is an FDA approved process that facilitates thedevelopment and expedites the review of drugs to treat serious diseasesand fill an unmet medical need with the goal of getting important newtreatments to patients earlier. This process allows a company to filethe sections of the NDA as they become available instead of filing allthe sections at once. It also enables the agency to commence its reviewand proceed on a rolling basis as the additional sections are completedand submitted for review.
Shire is completing a phase IIIclinical program that includes three phase III controlled studiesinvolving over 100 patients at 24 sites in 10 countries around theworld.
On July 6th, Shire announced that it filed a treatmentprotocol for velaglucerase alfa at the request of the FDA, which ifaccepted would allow physicians to treat Gaucher disease patients withvelaglucerase alfa on an early access basis, ahead of commercialavailability in the US. Under the conditions of the treatment protocol,Shire would provide velaglucerase alfa free of charge initially, inorder to provide access to patients as quickly as possible.
Velaglucerasealfa is made with Shire’s proprietary technology, in a human cell line.The enzyme produced has the exact human amino acid sequence and carriesa human glycosylation pattern.