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A new initiative from the US Food and Drug Administration (FDA) aims to develop “an optimized, fit-for-purpose” framework to facilitate the development and marketing of treatments for rare diseases, according to a 14 October announcement from the agency.

The US Food and Drug Administration (FDA) on Wednesday finalized guidance addressing frequently asked questions related to the development of cellular and gene therapy (CGT) products.

The pharmaceutical industry has largely endorsed the US Food and Drug Administration's (FDA) guidance on next-generation sequencing (NGS) for assessing the safety of human gene therapy products. However, they have requested clarification on several points, including off-target data, nomination methods, and additional information on how to assess these products following any manufacturing changes.

The US Food and Drug Administration (FDA) roadmap to reduce animal testing as a requirement for drug development has already made an impact after its first year, but the initiative faces future challenges in scaling and international harmonization, a group of experts wrote in a recent paper published in Stem Cell Research & Therapy.

As cell and gene therapies (CGTs) continue to advance, they have unique developmental features that may require innovation in a clinical trial setting, the authors of a recent paper explained.

The US Food and Drug Administration (FDA) has proposed guidance on the types of scientifically valid prior knowledge sponsors should consider when developing certain gene therapies (GT). The agency said the recommendations in the guidance are intended to improve review efficiency and help accelerate product development.

The US Food and Drug Administration (FDA) has released a final guidance document with immediate effect advising sponsors on chemistry, manufacturing, and controls (CMC) flexibilities for cell and gene therapy (CGT) products developed for biologics license applications (BLAs).

The US pharmaceutical industry expressed support for the US Food and Drug Administration’s (FDA) proposed framework for increasing access to treatments for ultra-rare diseases yet raised questions concerning various aspects of the program.