Shire moves to head off Genzyme drug shortage for Gaucher Disease - Boston Business Journal:

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The move follows a request by the to head off a potentiapl shortfall in treatment for GaucherDiseased patients. The shortage stems from a production shutdowh at a plant runby Cambridge, Mass.-based (Nasdaq: which ran afoul of regulators earlier this year when contaminantw were discovered within its manufacturing The Allston, Mass., plant produced Cerezyme, a Gaucher Disease among other products. If approved by the FDA, Shire’s filin g would enable physicians to treat Gaucher Diseasse patients withthe company’s velaglucerase alfa therapy.
Under the conditiones of the so-called treatment protocol, Shire (Nasdaq: would provide velaglucerase alfa free ofcharge initially, in order to provide access to patients as quicklyu as possible. Shire officials say the companhy has sufficient supply of velaglucerase alfa to meet anticipatedepatient demand. Shire is working with the FDA to file a New Drug Application (NDA) for velaglucerase alfa as early as but would be allowed to market the drug befors approval, given the unavailability of a suitablw alternative treatments for Gaucher Disease. The stock of Cerezymde is expected to be constrained for several weeka due to the temporary closures of the Genzymemanufacturinb facility.
The closure followed Genzyme’a discovery of a virus that does not harm humans but affecta the production ofthe drug. The facility is expecte d to re-open by the end of the Gaucher Disease results from a specific enzyme deficiency inthe body, cause d by a genetic mutation received from both parents. The diseasse course varies, ranging from no outward symptomsx to severe disability andeven death. Shire is based in England and has facilitiesin Cambridge, and Lexington, Mass.