FDA Announces Plans for Advancing Cell and Gene Therapies

In anticipation of a surge of investigational new drug applications for cell and gene therapies in the next few years, FDA Commissioner Scott Gottlieb and CBER Director Peter Marks announced plans to issue new guidance to advance their development.

The FDA intends to release multiple clinical guidances on different areas of products being actively developed, they said, including guidance on developing gene therapies for inherited blood disorders like hemophilia.

It also plans to issue guidance clarifying how the accelerated approval pathway may be used when a gene therapy treatment’s target is an underlying monogenetic change that causes a serious disorder not addressed by available therapy. In such cases, the gene therapy might “offer the potential to alter or cure the underlying genetic defect that gives rise to, and causes the advance of, a disease,” they said.