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Funding drugs for rare diseases
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Shire plc has submitted an application for approval to European regulators for its drug target, velaglucerase alfa. The drug candidate, for Gaucher disease, is an alternative to Genzyme Corp.’s Cerezyme. Regulators in both the U.S. and Canada have already fast-tracked the drug to help head off a shortage of Cerezyme, due to the temporary closure of a Genzyme plant in Allston.
The UK-based company, whose Human Genetic Therapies division is headquartered in Cambridge and Lexington, said that European regulators have also agreed to speed up the approval process for the drug target. The European review will begin in December and will take 150 days, down from the usual 210 days.
Patients in Europe and other regions outside the U.S. are already receiving the drug candidate under emergency pre-approval access programs designed specifically to address the continuing supply shortage. In the U.S., patients continue to be enrolled in an FDA-approved treatment protocol that has been open since September 2009.
The shortage followed the discovery of a virus at Genzyme’s Allston manufacturing plant, which forced a six-week closure of the facility and the dumping of some unfinished batches of Cerezyme. The drug is currently being directed toward the sickest patients.
Shire’s drug target would treat the most common form of the disease, Gaucher type 1. Some of these patients are currently unable to receive Cerezyme.
Shire’s (Nasdaq: SHPGY) stock rose slightly on the news, to $58.46 in midday trading, up from $57.70 at the previous close.
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