The US Food and Drug Administration intends to guard against rewarding “trivial” changes between two gene therapy products with orphan drug designation and seven-year orphan exclusivity, Office of Tissues and Advanced Therapies director Wilson Bryan said on 2 April.
Gene Therapies: ‘Trivial’ Changes Will Not Be Rewarded With Orphan Drug Designation And Exclusivity
For two gene therapies with the same transgenes and/or vectors but different promoters, the US FDA’s ‘sameness’ determination under the orphan drug regulations will consider whether that difference has any impact on product activity, the agency's Wilson Bryan says.
