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

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 that the FDA really had no choice but to grant the anti-viral remdesivir an orphan designation for its use in coronavirus infection because at filing the disease had not infected more than 200,000 people, the threshold for rare disease status

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

More from Rare Diseases

More from Pink Sheet