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.
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Gene Therapies: US FDA Clarity Needed On ‘Sameness’ Determinations For Products From Same Vector Class
Agency also should better define, and provide examples of, the types of minor differences and additional features that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.
CBER’s heavy COVID-19 workload is pushing other things to the side, causing delays in sponsor meetings and issuance of new guidance documents, and a slowdown in efforts to streamline development of individualized therapeutics, biologics center leaders tell BIO Digital 2020.
US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final product’ in making the determination.