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Orphan Exclusivity For Gene Therapies Hinges On Two Big Factors

Executive Summary

US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final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.

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US FDA Tweaks Guidance On Sameness Of Gene Therapies, But Questions Remain

In response to industry input, the agency made a few changes to its draft guidance but several issues remain uncertain, such as what constitutes ‘minor differences’ between gene therapy products.

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that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.

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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