Gene Therapies: ‘Trivial’ Changes Will Not Be Rewarded With Orphan Drug Designation And Exclusivity
Executive Summary
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.
You may also be interested in...
Bluebird’s Lost Voucher: Lyfgenia Contains Same ‘Active Ingredient’ As Zynteglo, US FDA Says
Surprise denial of rare pediatric disease priority review voucher could prompt calls for the agency to re-examine how it interprets the term ‘active ingredient’ for gene therapies. Bluebird asserts there are significant differences between its gene therapies for treating sickle cell and beta-thalassemia.
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that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.
US FDA’s Gene Therapy Work Feeling The Effects Of Pandemic Response
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.