Orphan Exclusivity: Rarely Used, But Well-Known, Loophole Would Be Closed By House Legislation
US FDA could revoke orphan designation or exclusivity if cost recovery requirements were not satisfied under bill unanimously passed by the House Energy and Commerce Committee.
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Restricting orphan drug approvals could save the US billions of dollars, study from University of California’s Center for Health Policy and Economics suggests.
Rare Pediatric Disease Designations May Be Unavailable After July As Program Renewal Remains Uncertain
Without new legislation, US FDA said that designations, which are required to receive priority review vouchers, cannot be assured if requested after July. While the window for designations formally closes at the end of September when the authorization of the program expires, the agency is telling sponsors that it needs 60 days before that to review any requests. If a product does get a designation, it would need to secure approval by 30 September 2022 to gain a priority review voucher.
Two days after US FDA granted remdesivir orphan status for treatment of COVID-19, Gilead told the agency it is unnecessary. EMA says it would not grant orphan drug designation to a COVID-19 treatment.