FDA approaches Shire and Protalix to fill Gaucher drugs void

Shire has been asked to submit its investigational treatment for Gaucher disease to regulators in the USA as a result of a shortage of the approved drug for the condition, Genzyme Corp's Cerezyme.

The UK drugmaker says that it filed a treatment protocol for its enzyme replacement therapy velaglucerase alfa, at the request of the US Food and Drug Administration, “in view of a potential restriction on the availability” of Cerezyme (imiglucerase). Last month, Genzyme temporarily shut down its manufacturing facility in Boston after a bioreactor was contaminated with a virus, affecting production of Cerezyme and Fabrazyme (agalsidase beta), which is indicated for Fabry disease.

Shire noted that if approved by the FDA, the treatment protocol would allow doctors to treat Gaucher disease patients with velaglucerase before it becomes commercially available. The company said it would provide the treatment “free of charge initially, in order to provide access to patients as quickly as possible” and is currently working with the FDA to file a New Drug Application.

The FDA has also approached Israeli-headquartered Protalix BioTherapeutics and asked the company to consider submitting a treatment protocol for its Gaucher disease treatment prGCD (glucocerebrosidase), which is in Phase III. Protalix, which will also provide its drugs free of charge, expects approval of the treatment protocol within the next 60 days.