Gene therapy has broken out of its chains. In 2017 alone, Kymriah (Novartis), Luxturna (Spark), and Yescarta (Kite Pharma/Gilead) received FDA approval. The number of small gene therapy companies is rapidly expanding and large biopharma companies like Amgen, BioMarin and AbbVie are getting heavily involved in their own gene therapy projects. AbbVie partnered up with Voyager Therapeutics to use gene therapy to treat Alzheimer’s and other nuerodegenrative diseases. BioMarin is working on an AAV-factor VIII vector to treat hemophilia and Amgen bought stakes in several smaller gene therapy companies to push forward its CAR T and RNA Interference programs. These are just three examples, but there are dozen more just like them. I would venture to say that every large biotech company has considered, in some form or another, expanding into the gene therapy space.
In July, 2018 the FDA Commissioner, Scott Gottlieb released a statement outlining the agencies efforts to advance the development of gene therapies. In the statement Scott Gottlieb recognized the rapid expansion of gene therapy advancements and the unprecedented approval of three gene therapy products in 2017. He also states that the new steps taken by the FDA are designed to foster the development of innovation within the gene therapy field. The statement implies that in some cases gene therapy products can be accelerated through the approval process by using what is known as phase 4 confirmatory trials or the post-marketing clinical trials. The statement also announced a suite of six regulatory draft guidance documents designed to provide clear recommendations to sponsors and researchers.
FDA Gene Therapy Draft Guidances
- Human Gene Therapy for Hemophilia
- Human Gene Therapy for Retinal Disorders
- Human Gene Therapy for Rare Diseases
- Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)
- Testing of Retroviral Vector-Based 1 Human Gene Therapy Products for 2 Replication Competent Retrovirus 3 During Product Manufacture and 4 Patient Follow-up
- Long Term Follow-Up After Administration of Human Gene Therapy Products
PBL can help with many of the tests outlined in the FDA guidance documents above. Please contact us to learn how we can help with your gene therapy program.