Fabry Disease Patients Get Sicker as Drugs Go Overseas


"We provide the drug to every patient in the world that needs it," countered Genzyme's Piela. "It's an extremely rare disease, and we have been treating patients in Europe, Latin America and Asia where it's been approved.

In addition to asking the FDA to stop overseas shipments of the drug, the plaintiffs filed petitions with the National Institutes of Health to allow other manufacturers to license the patent on Fabrazyme. That was denied in December.

Patients could also ask for an executive order by President Obama. "This is extraordinarily rare and used for national defense, but I think it rises to that level," said Black.

Walter said he agrees that critical action is needed.

"It's not really fair to accuse [Genzyme] of wrongdoing for a virus, but they could have planned better and built an inventory," he said. "But now, we are not confident they are doing everything they can for the Fabry community."

His last infusion was set for Aug. 16 and never arrived; as his Aug. 30 dose approaches, Walter still doesn't know if he will be able to get it.

He called Genzyme, but said, "It's been a good two weeks with no answers yet. I am about to miss my second one in a row, and I don't know what's happening. ... We are in grave need of help."

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