The US Food and Drug Administration (FDA) last week finalized guidance to assist sponsors developing human gene therapy (GT) products for neurodegenerative diseases. In the final version, FDA acceded to industry’s requests to modify its language on the use of crossover trials, provided more detail on the use of comparability studies in assessing the effect of post-approval manufacturing changes on products and eliminated limits for residual product-related impurities.
The document finalizes a draft version issued in January 2021. (RELATED: FDA guides on Covid considerations in cell and gene therapy, Regulatory Focus 20 January 2021)
Industry stakeholders requested changes to the draft guidance in comments submitted to the public docket. (RELATED: Drugmakers suggest tweaks to FDA’s neurodegenerative disease gene therapy guidance, Regulatory Focus 6 April 2021)…
