Print
Email
Print Edition Stories
MHT's Galen Moore: Hiring trends and opportunities
MHT's Marc Songini: Technology to keep seniors healthy
MHT's Marc Songini: Researchers focus on macular degeneration
Shire plc (LSE: SHP, NASDAQ: SHPGY) has filed a treatment protocol with the U.S. Food and Drug Administration for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher Disease.
The move follows a request from the FDA, in view of a potential restriction on the availability of Cambridge-based Genzyme drug Cerezyme, the current approved and marketed treatment for Gaucher Disease patients.
If approved by the FDA, the treatment protocol would allow physicians to treat Gaucher Disease patients with velaglucerase alfa ahead of commercial availability in the U.S. 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.
Shire officials say the company has sufficient supply of velaglucerase alfa to meet anticipated patient demand.
Shire is working with the FDA to file a New Drug Application for velaglucerase alfa as early as possible, but would be allowed to market the drug before approval, given the unavailability of suitable alternative treatments for Gaucher Disease.
The stock of Cerezyme is expected to be constrained for several weeks due to the temporary closure of a Genzyme manufacturing facility in Allston. The closure followed Genzyme’s discovery of a virus that does not harm humans but affects the production of the drug. The facility is expected to re-open by the end of July.
On Monday, Israel-based Protalix BioTherapeutics Inc. said it had been contacted by the FDA about possibly initiating a treatment protocol for its own drug target for Gaucher Disease.
Gaucher Disease results from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. The disease course is quite variable, ranging from no outward symptoms to severe disability and death.
Shire is based in England and has facilities in Cambridge and Lexington.
RSS Feeds
Comments
Please Login/Register to post comments.
No comments have been added or approved.