The FDA has proposed a framework to accelerate gene therapy development by allowing manufacturers to rely on existing scientific knowledge, GxP News reports.
The US Food and Drug Administration has proposed allowing cell and gene therapy manufacturers working on rare and life‑threatening diseases to use existing scientific knowledge to speed up development. The guidance is intended to cut gene therapy development time, which is particularly important for patients with rare diseases.
The FDA document explains that developers may draw on previously accumulated chemistry, manufacturing and control data, as well as preclinical and clinical information obtained from developing other similar products. Such prior knowledge includes medical and scientific data recognised by the expert community, as well as experience in developing and manufacturing similar drugs and processes.
Using this data does not mean lowering standards, the agency said. However, it believes this will streamline the submission process and subsequent review, ultimately speeding up the market launch of new products.
Developers must provide a scientific rationale demonstrating that the data they are using apply to their specific product and development context. The FDA encourages early engagement, including through pre‑investigational new drug application meetings.
Earlier this year, the FDA proposed a new mechanism for accelerated approval of personalised treatments for rare genetic diseases, allowing manufacturers to rely on small, well‑controlled studies when traditional clinical trials are not feasible. The agency also launched an initiative to repurpose already approved drugs for new uses, aiming to speed patient access to effective treatment options.
The agency faced intense scrutiny under former commissioner Marty Makary, who stepped down last month. His departure followed a series of controversial decisions on rare disease treatments and public conflicts with drug manufacturers. A permanent successor has not yet been named; deputy commissioner for food, Kyle Diamantas, is serving as acting commissioner.
