FDA Releases New Info About How it Could Regulate Gene Therapy

According to a story from finance.yahoo.com, the US Food and Drug Administration (FDA) has recently released new guidelines that are related to how the agency will regulate and manage the development of gene editing drugs. Gene therapy is an approach to medicine that is undoubtedly starting to pick up steam, and the FDA’s new set of goals will help facilitate how these therapies will be developed.

First and foremost, the agency is making it clear that Accelerated Approval will be a potential option for some gene therapies. This applies more specifically to therapies that will be developed as a single use therapy. These types of gene therapies typically insert a functional gene to completely replace the mutated gene that causes disease symptoms. Some companies that could potentially benefit from this new incentive include the gene therapy Zolgensma, which is currently being tested by Novartis. This therapy is intended as a treatment for spinal muscular atrophy type 1. Another example is an experimental gene therapy being developed by Sarepta Therapeutics as a treatment for Duchenne muscular dystrophy.

Other gene therapies will be expected to continue down the more conventional development path. The agency specifically noted that therapies that intend to treat a genetic disease by initiating the production of proteins that play a role in a disease should expect the therapy’s development to continue at the standard pace.

The FDA also appears ready to give a helping hand to the developers of CAR-T cell therapies. These drugs are used to treat cancer; while not commonly referred to as a gene therapy, the process of treatment with these therapies involves the extraction of a cancer patient’s T-cells, which are then genetically modified to produce a specific T-cell receptor which will allow them to recognize and attack cancer cells. The agency hopes to help introduce more effective methods for manufacturing CAR-T cell therapies; the FDA states that it is willing to accept real-world data (data outside of clinical trials) for these types of therapies after manufacturing changes have been made.

While these approaches haven’t been made official yet, it is likely that it is only a matter of time. The FDA will soon be dealing with a lot of potential gene therapy candidates; by 2025, the agency expects somewhere between 10 and 20 new gene therapies to be approved per year.


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