Six New FDA Guidance Documents Open for Comment

ASGCT Staff - July 19, 2018

FDA infographic by Michael J. Ermarth

UPDATE: On Sept. 6, 2018, "in response to requests for an extension to allow interested persons additional time to submit comments and any new information," the Food and Drug Administration extended the deadline for written and electronic comments to Dec. 10, 2018.

With new FDA guidance drafts addressing hemophilia, retinal disorders, rare diseases, and manufacturing standards, ASGCT will be evaluating the specific content of the guidance documents leading up to the September 13 liaison meeting with the FDA. The Society will provide recommendations related to the details of these guidance documents.

“Once just a theory, gene therapies are now a therapeutic reality for some patients,” FDA Commissioner Scott Gottlieb, MD, said in a July 11 statement following the release of the FDA's much-anticipated six guidance documents on gene therapy. Reflecting the rapid advancement in the field, three of them are the first for disease-specific applications of gene therapy—for hemophilia, retinal disorders, and other rare disease. All six FDA guidance documents are open for comment on Regulations.gov through October 10, 2018.

The information is intended to assist sponsors in addressing the challenges of rare disease clinical development, such as limited study population size. In addition, the draft guidance on gene therapy for treatment of hemophilia provides recommendations regarding surrogate endpoints that could be used by sponsors pursuing accelerated approval of such products.

The three disease-specific guidance documents:

Human Gene Therapy for Hemophilia

Human Gene Therapy for Retinal Disorders

Human Gene Therapy for Rare Diseases

The other three guidance documents, which provide sponsors with manufacturing recommendations, are updates on previous guidance documents in need of revision due to the rapid advances in the field since their original release.

These documents offer guidelines to sponsors regarding the provision of sufficient chemistry, manufacturing and control (CMC) information to assure safety, identity, quality, purity and strength/potency of gene therapy products; the proper testing for replication competent retrovirus during the manufacture of retroviral vector-based gene therapy products; and the design of long-term follow-up (LTFU) observational studies for the collection of data on delayed adverse events following administration of a gene therapy product.

The three manufacturing and safety guidance documents:

Chemistry, Manufacturing, and
Control (CMC) Information for
Human Gene Therapy Investigational
New Drug Applications (INDs)

Long Term Follow-Up After
Administration of Human
Gene Therapy Products

Testing of Retroviral Vector-Based
Human Gene Therapy Products for
Replication Competent Retrovirus
During Product Manufacture and
Patient Follow-up

Comments are due on all guidance documents by October 10, 2018. ASGCT will be evaluating the specific content of the guidance documents over the course of the comment period and leading up to its liaison meeting with the FDA on September 13, and will provide Society recommendations related to the details of these guidance documents.

ASGCT appreciates the level of attention that FDA is providing to gene therapy at this important turning point in its development.

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