Nature Medicine | Spoonful of Medicine

Patient groups cautiously optimistic about Fabrazyme plant approval

Two-and-a-half years after a virus struck Genzyme’s main production plant—triggering worldwide shortages of Fabrazyme, an enzyme replacement therapy for Fabry’s disease, an illness that afflicts fewer than 10,000 people worldwide—regulators in the US and EU have approved a new manufacturing facility. With the opening of the new plant, Genzyme announced today that all Americans currently taking Fabrazyme (agalsidase beta) and the most severely affected European patients should be up to full dosing by the end of March.

“This is extremely encouraging and exciting news,” says Jack Johnson, executive director and cofounder of the Fabry Support and Information Group, a Missouri-based advocacy organization for patients with the rare inherited disorder that causes fatty substances to build up in the eyes, kidneys, nervous system and cardiovascular system, leading to pain and organ complications. “FDA approval of that plant is something that the patient community has been waiting on for a long time.”

Genzyme now plans to shift production of Fabrazyme entirely to the newly approved plant in Framingham, Massachusetts, leaving its Allston, Massachusetts plant—the site of contamination with a vesivirus in its bioreactors in June 2009—to focus on the company’s Gaucher’s drug Cerezyme (imiglucerase).

The company, which was acquired last year by the French drug giant Sanofi, also said today that it will begin transitioning new US patients onto full doses of Fabrazyme, although a timeline was not given. That plan is too slow for C. Allen Black, a Pennsylvania lawyer who, in addition to representing plaintiffs who have been denied access to Fabrazyme or suffered injuries on reduced dosages, is also leading a citizen’s petition demanding that the US Food and Drug Administration prioritize domestic drug supplies ahead of foreign markets. “American taxpayers paid for the invention,” he says. “So it doesn’t make any sense that they’ve triaged Europeans ahead of Americans.”

Black also represents a group of people with Fabry’s who petitioned the US National Institutes of Health (NIH) to employ the government’s so-called ‘march-in’ rights and demand that Genzyme allow other companies to make Fabrazyme in the face of such life-threatening drug shortages. The NIH denied the request in late 2010, and, according to Black, the agency has not yet responded to an appeal filed in April of last year. Although the pace of the march-in process continues to frustrate Black, he stands by the need to break the Genzyme patent, even with the company’s improved manufacturing capacity. “I still firmly believe that we need a reliable source of Fabrazyme, and just because Framingham is online doesn’t mean this won’t happen again,” he says.

Jerry Walter, president of the Washington, DC-based National Fabry Disease Foundation, similarly hedges in response to today’s announcement. “We’re very happy with the approval,” he says. But he would still like to see the FDA approve Replagal (agalsidase alpha), a rival enzyme replacement therapy from UK-based Shire that is currently available in the EU but not the US, so that patients have a back-up drug should shortages ever strike again. The opening of the Framingham plant “makes us feel more comfortable that we’re going to be able to go back to normal and get full dose of medication,” he says, “but after everything we’ve been through we want more options as well.”

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