Nature Medicine | Spoonful of Medicine

NIH faces marching orders on orphan drug shortage

fabcrop.jpgBy Elie Dolgin

Ever since a virus contaminated Genzyme’s production plant in Allston, Massachusetts close to two years ago, people with Fabry’s disease have faced severe shortages of the medicine they need, Fabrazyme (see editorial on page 515). In August 2010, three people with Fabry’s petitioned the US National Institutes of Health (NIH) to step in and demand that Genzyme allow other companies to make the enzyme replacement therapy on the basis of the government’s so-called ‘march-in’ rights. The provision of the Bayh-Dole Act allows funding agencies to override exclusivity rights to intellectual property arising from government-funded research when people’s lives are at risk.

The NIH denied the request late last year. But, given Genzyme’s continuous production delays—the company now says it won’t be manufacturing Fabrazyme again until closer to the end of the year—on 5 April the petitioners appealed the original decision.

Such requests have historically failed. But this time could be different.

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