- Food and Drug Administration Commissioner Scott Gottlieb announced a "modern, comprehensive framework" for development, review and approval of gene therapies, which aims to "advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness."
- The blueprint started this week with issuance of three new draft guidance documents on the development of gene therapies for specific disease categories: hemophilia, retinal disorders and rare diseases.
- The FDA has also issued comprehensive updates to three existing sets of guidance: Chemistry, manufacturing, and control for investigational drug applications; testing of retroviral vector-based gene therapy products for replication competent retrovirus during product manufacture and patient follow-up; and long term follow-up after administration of human gene therapy products. These updates will replace guidance issued in 2006 and 2008.
Gene therapies have potential to treat or even cure serious and life-threatening diseases. Three such treatments have reached the U.S. market over the past year, but the newness of this approach to treatment and difficulty in gaining full data on safety, efficacy, manufacturing quality and long-term impact in pre-market trials is causing approval challenges.
Gottlieb previewed the gene therapy policies at a fireside chat at the BIO International convention last month. He said that July would bring six new sets of guidance to lay out what he described as the "modern framework" of the agency's approach to gene therapies.
This suite of six scientific guidance documents is the most recent in a pipeline of changes targeting innovative therapies, including cell and gene therapies, in the U.S. The changes began with a comprehensive policy framework to provide a lower-cost and more streamlined approach to approval for cell therapy products and procedures, put in place in August 2017. This included the breakthrough therapy designation, and the regenerative medicine advanced therapy designation.
These guidance documents show the FDA's support for innovative products, and should be positive for investors.
"From an investment standpoint, we think the documents suggest FDA willingness to be flexible in the design of trials and endpoints and to accept some level of uncertainty around some [gene therapies] prior to the time of approval [such as] long-term durability and safety."
Leerink analyst Joseph Schwartz also saw this as positive, particularly for companies in this field.
"Our optimism on gene therapy for its potentially curative prospect is supplemented by FDA's generally favorable [and] lenient stance under Commissioner Gottlieb. Our review of the recent draft guidances… continues to suggest FDA's unwavering willingness to adopt novel technologies addressing unmet medical needs," he wrote in a note to clients.
Schwartz said the firm predicted "positive implications for rare disease-focused gene therapy being developed by the likes of [Audentes Therapeutics, BioMarin Pharmaceutical, UniQure, Ultragenyx Pharmaceutical, Solid Biosciences, and Sarepta Therapeutics]."