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Remdesivir’s Path To Orphan Designation Could Be Exploited By Others, Experts Warn

 
• By Derrick Gingery

Congress should give FDA more flexibility to administer orphan designations, such as the ability to revoke designations later if the disease prevalence grows beyond the rare disease threshold.

Orphan Drug_1200
If the loophole is not closed, more sponsors could follow the pathway that allowed remdesivir to receive an orphan drug designation as a coronavirus treatment, the writers of a JAMA Internal Medicine article argued. • Source: Shutterstock

Gilead Sciences, Inc.’s short flirtation with orphan status for remdesivir could be a valuable lesson that the US Food and Drug Administration needs more flexibility to administer the orphan drugs program, including the ability to revoke designations later for cause.

In a recent viewpoint in JAMA Internal Medicine, Kao-Ping Chua of the University of Michigan Medical School, and Rena Conti, of the Boston University Questrom School of Business, argue...

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