Fabry Disease Patients Get Sicker as Drugs Go Overseas

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"This story should scare the hell out of everyone in America," said Black, the lawyer. "There's a lot of monopoly out there. It could have hit insulin production or chemotherapy."

In all, 18 members of Walter's family have been affected by the X-linked genetic disorder. Men with the gene can pass it on to their daughters. Women with the gene have a 50-50 chance of passing it on to their sons or daughters.

The average age of death for an untreated male is 50, according to Walter, whose brother died at 37. He said the drug had been a godsend, but now Fabry patients feel abandoned.

"I was down to a third of a dose at one point, and now I am back to a half, but recently the shipments have been on hold completely with no expected date to return to shipping," said Walter.

Genzyme's Piela said that despite delays, August doses will be available to patients soon. "However, we do not expect to ship Fabrazyme to meet this allocation until September," he said.

"These are biologic drugs, and there is variability in production based on various factors," said Piela.

The company is in the midst of constructing a second manufacturing plant in Framingham, Mass., and when that is approved "We will be able to meet full demands," he said. "But licensing takes time."

Piela said he expects production there to be in full swing by 2012 and "significantly increase capacity and help end the supply shortage."

But that isn't soon enough for patients who say they need full dosages of the drug now.

"There are really no options," said one 27-year-old New Yorker with the disease, who did not want his name used because the Fabry community is so small he fears he might not get his medication.

"Recently, [Genzyme] canceled another dose for me -- the appointment was for one of two people. I don't know if they are manually picked, and I would rather not give them a reason not to pick me again. ... I don't want to be singled out as a troublemaker." His last half dose was Aug. 3.

Now, he is considering moving to France, where he has dual citizenship and can obtain the lifesaving Fabrazyme or Replagal.

"I count my blessings," he said. "Thankfully, there's a treatment option out there."

But American patients still want to know why they cannot have the same access to full doses as patients in Europe. Genzyme officials said that the European Medicine Agency has required full dosages of Fabrazyme, and the company is complying with that "guidance."

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