CBER Announces 2020 Guidance Agenda

FDA’s Center for Biologics Evaluation and Research (CBER) announced the list of guidance documents it plans on publishing in 2020 that includes nine documents focused on gene therapies. CBER is also planning on guidance for chimeric antigen receptor (CAR) T cell therapies; clinical trial designs; and chemistry, manufacturing, and controls changes, in addition to guidance documents on blood and blood components.

The following are the guidance documents that will address gene therapies:

• Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-Up; Guidance for Industry (Issued January 2020)

• Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry (Issued January 2020)

• Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications; Guidance for Industry (Issued January 2020)

• Human Gene Therapy for Hemophilia; Guidance for Industry (Issued January 2020)

• Human Gene Therapy for Retinal Disorders; Guidance for Industry (Issued January 2020)

• Human Gene Therapy for Rare Diseases; Guidance for Industry (Issued January 2020)

• Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Draft Guidance for Industry (Issued January 2020)

• Human Gene Therapy for Neurodegenerative Diseases; Draft Guidance for Industry

• Considerations for the Development of Human Gene Therapy Products Incorporating Genome Editing; Draft Guidance for Industry

Source: FDA