Drugmakers suggest tweaks to FDA’s neurodegenerative disease gene therapy guidance

Several drugmakers and Health Canada are suggesting changes to the US Food and Drug Administration’s (FDA) recently released draft guidance on developing gene therapies for neurodegenerative diseases.

Released in January, the draft guidance provides recommendations for the development, testing and clinical study of gene therapies for neurodegenerative diseases. The guidance also discusses considerations for chemistry, manufacturing and controls (CMC) and offers an overview of the regulatory pathway and opportunities for engagement with FDA for such products.