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that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.
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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.
Orphan Exclusivity For Gene Therapies Hinges On Two Big Factors
US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final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.
Merck & Co. Files First IRA Suit, Signaling Industry Is Ready To Fight
Coming less than three months before the statutory deadline for CMS to publish the initial list of drugs, Merck’s lawsuit calls US Medicare drug price negotiations ‘extortion’ in a complaint that could be the first of more legal pushback to follow.