Exclusive: FDA issues industry-friendly gene therapy guidance
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The Food and Drug Administration on Tuesday will issue draft guidance for how companies developing cell and gene therapies can rely on existing scientific knowledge to avoid unnecessary tests and speed up the development process, the agency exclusively told Axios.
Why it matters: The move could be seen as an olive branch to the drug industry and the rare disease community in the wake of former commissioner Marty Makary's departure. But it still will all come down to how the guidance is implemented.
The growth of cell and gene therapy has expanded the knowledge base for treating diseases at the molecular level and developing certain products and processes.
- The new FDA guidance outlines how this prior knowledge can be used so that companies aren't essentially starting from scratch each time they develop a new product.
- Using prior knowledge "may be particularly helpful in the context of [genome editing] products intended to treat rare diseases, many of which may be serious and life threatening," the FDA writes.
Between the lines: Under Makary's tenure, the FDA had a particularly tumultuous relationship with the rare disease community and small biotechs attempting to bring cell and gene therapies to market.
- The industry complained that the agency repeatedly went back on what it had previously told companies about drug reviews and standards.
- And while FDA officials publicly spoke of regulatory flexibility and a vision of bringing new therapies to market faster, the agency's real-world requirements of specific companies were frequently accused of being unnecessarily onerous.
The big picture: The Trump administration is searching for a new FDA commissioner while dozens of other leadership positions at the agency also remain vacant.
- The direction the agency takes has huge financial implications for the drug and biotech industries, as well as patients themselves.
- And all of this is playing out against China's rapid rise as a biopharmaceutical power — and calls for the U.S. to revamp its regulatory approach to stay competitive.
