With industry's blessing, FDA launches novel excipient review pilot program
The FDA on Tuesday launched a new pilot program that will initially review four novel excipients over two years, with an eye toward helping drug developers in situations where existing excipients may be difficult to employ.
The plan for such a pilot has been in the works since at least 2019, as the FDA does not currently review new excipients as part of an IND or a marketing application, although some novel excipients may provide public health benefits, such as improved drug delivery or potentially abuse-deterrent properties that could be key for new opioid formulations.
FDA, which defines “excipient” as any ingredient intentionally added to a drug but not intended to have a therapeutic effect, received support from top biopharma companies like Regeneron, Janssen and AstraZeneca in its plan to craft such a pilot, noting the current lack of excipient innovation.
AstraZeneca pointed out:
The limited number of approvals containing novel/semi-novel excipients in the US and elsewhere in recent years speaks to the current incentives (or lack of them). While changes in the development pipeline may start to drive alternative models, changes to the current model with respect to excipients could also change the development pipeline, for example making currently unattractive and/or undruggable candidates more viable.
So how will the new pilot work? Initially, the program will be available for novel excipients that (1) have not been previously used in FDA-approved drug products, and (2) do not have an established use in food.
In the first part of the program, manufacturers have until Dec. 7 to provide a high-level overview of their novel excipient, including brief summaries describing the novel excipient, its proposed use, and the public health or drug development need addressed by the excipient.
Four excipients will then proceed to the second part of the pilot, where manufacturers will submit to the FDA more extensive toxicology, pharmacology and other carcinogenicity data, according to the Federal Register.
“A determination that the excipient is appropriate for use in clinical trials means that FDA has determined it is appropriate to use the novel excipient in an IND within the defined use without additional justification,” the FDA says. “However, the drug sponsor would still need to demonstrate that the excipient is safe in the proposed formulation.”