Regulatory Oversight On Gene Therapy In The U.S. And EU

Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for lymphoma have ushered in a new era of therapeutics.

With the rapid evolution of the gene therapy field, regulatory agencies have been working to keep pace with these scientific and clinical breakthroughs.

An understanding of the regulations and guidance documents reflecting regulator current thinking surrounding gene therapies is essential to success, and consideration of the regulatory expectations and requirements at the earliest possible stage of development will increase the likelihood that these products will ultimately enter clinical trials that will result in approved marketing applications and improvement in human health and welfare.