File this under 'if at you first you don't succeed...' After being rejected last month by the National Institutes of Health in a bid to override the patent on a Genzyme drug that is in perpetually short supply, a pair of Fabry's disease patients are now petitioning the FDA to insist that overseas stock of the med is first made available to US citizens.
The move comes amid ongoing difficulties Genzyme has encountered in producing sufficient supplies of Fabrazyme due to severe manufacturing problems that began in 2009. These prompted a $175 million fine, a consent decree and a takeover bid from Sanofi-Aventis. Existing patients, meanwhile, are subject to inadequate rationing and newly diagnosed patients are unable to receive any meds.
To rectify the situation, some patients last summer asked the US Department of Health and Human Services last summer to override the use patents held by Genzyme on its med, which is the only such treatment currently available for a rare, inherited disorder that causes kidney and heart problems. Last month, though, the NIH turned them down (back stories here and here).
Now, the patients want the FDA to level a playing field in which they say 62 percent of Fabrazyme made in the US is allocated to overseas patients, even though those patients have access to an alternative, Replagal, which is made by Shire Pharmaceuticals. While the European Medicines Agency recommended Fabry's patients switch drugs, Shire is not currently seeking approval to sell Replagal in the US.
"The result is the tragically ironic situation that there is no drug shortage overseas," Allen Black, the lawyer for the patients, writes us. "There have already been three deaths reported in the US under the rationing, but none reported overseas. Even more bizarre, is that the FDA is importing Replagal to replace the Fabrazyme that was shipped overseas in the first place. However to receive treatment, the patients must show 'critical medical need' on an individual basis."
And so the petition sent to the FDA asks the agency to allocate Fabrazyme first to US patients and, in the event the request isn't granted, that patients be allowed to present their case for medical need before Fabrazyme is sent overseas (you can read the petition here). Is this reasonable? What do you think the FDA should do?
Should the FDA Grant This Request?
- Yes (87%, 92 Votes)
- No (13%, 14 Votes)
Total Voters: 106