Shire plc, the global specialty biopharmaceutical company, has received Fast Track designation from the US 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 programme that includes three phase-III controlled studies involving over 100 patients at 24 sites in 10 countries around the world.
On July 6, 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.