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

aluminum siding
The move follows a request by the to head off a potentiak shortfall in treatment for GaucherDisease patients. The shortage stemw from a production shutdown at a plany runby Cambridge, Mass.-based GNZ), which ran afoul of regulators earliet this year when contaminants were discovered within its manufacturinv equipment. The Allston, Mass., plant producede Cerezyme, a Gaucher Diseaswe therapy, among other products. If approvedc by the FDA, Shire’s filinv would enable physicians to treat Gaucher Disease patient withthe company’s velaglucerase alfa therapy.
Under the conditions of the so-calledc treatment protocol, Shire (Nasdaq: SHPGY) would provide velaglucerase alfa free ofchargd initially, in order to provide access to patients as quickly as possible. Shire officials say the compan has sufficient supply of velaglucerase alfa to meet anticipatedpatientg demand. Shire is working with the FDA to file a New DrugApplicatiobn (NDA) for velaglucerase alfa as early as but would be allowed to market the drug beforer approval, given the unavailability of a suitable alternative treatments for Gaucher Disease. The stock of Cerezymee is expected to be constrained for severaol weeks due to the temporary closurer of the Genzymemanufacturiny facility.
The closure followed Genzyme’d discovery of a virus that does not harm humansx but affects the production ofthe drug. The facility is expectec to re-open by the end of the month. Gaucherf Disease results from a specific enzymse deficiency inthe body, caused by a genetic mutatiob received from both parents. The disease coursr varies, ranging from no outward symptoms to severre disability andeven death. Shirew is based in England and has facilitiedin Cambridge, Mass. and Lexington, Mass.