Shire plc has announced that, at the request of the FDA, in view of a potential restriction on the availability of the current approved and marketed treatment for Gaucher’s disease patients, it has filed a treatment protocol for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher’s disease.
If approved by the FDA, the treatment protocol would allow physicians to treat Gaucher’s disease patients with velaglucerase alfa 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.
Shire has sufficient supply of velaglucerase alfa to meet anticipated patient demand.
Shire is working with the FDA to file a New Drug Application (NDA) for velaglucerase alfa as early as possible.