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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US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final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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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that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.

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