FDA’s novel excipient pilot program opens for candidates

Regulatory NewsRegulatory News | 08 September 2021 |  By 

The US Food and Drug Administration (FDA) has launched a new pilot program to review novel excipients for use in meeting unmet needs in formulating new drug products. The pilot has the full support of the pharmaceutical industry.
FDA called the program “a new pathway for manufacturers to obtain FDA review of certain novel excipients (inactive ingredients) prior to their use in drug formulations,” according to a 7 September announcement.
Excipients are inactive ingredients reviewed as a component of a finished drug product in a new drug application or a generic application, whereas a novel excipient is an inactive ingredient that has not been previously used in an approved drug product. Drug manufacturers may be reluctant to use novel excipients because it is unclear that FDA would accept the available safety information on the material and approve its use in a drug product.
The agency in the announcement acknowledged the “developmental challenges” that confront excipient manufacturers and drug developers in formulating products with certain excipients. Through this program, “FDA intends to foster development of excipients that may be useful in scenarios where excipient manufacturers and drug developers have cited prior difficulty in using existing excipients.”
The pilot program will be available for novel excipients that have not been previously used in FDA-approved drug products and do not have an established use in food, according to an outline of the program.
FDA will accept four initial proposals in the pilot “but will consider accepting more proposals as resources allow.”
FDA will use the following criteria to select candidates:
  • The potential public health benefit of the novel excipients; for example, such applications as use in opioid abuse-deterrent formulations or to promote new therapies for serious and life-threatening disease;
  • The likelihood of the novel excipient manufacturer’s ability to submit a complete package within the established timeframe; and
  • The potential of the novel excipient to “meaningfully improve pharmacokinetic characteristics” and lead to the development of novel drugs.
Excipient manufacturers whose initial proposals are accepted will then be asked to submit a full toxicology package as well as chemistry, manufacturing, and controls data in a process parallel to that for an investigational new drug application.
Biopharma companies Regeneron, Janssen and AstraZeneca, as well as the US Pharmacopoeia (USP) and the International Pharmaceutical Excipients Council (IPEC-Americas) all pledged their support for the program in public comments submitted last year. (RELATED:  Industry Supports FDA’s Plans to Review Novel Excipients Outside of Applications Regulatory Focus 4 February 2020).
Industry was responding to the agency’s calls for comments on whether to run this pilot. (RELATED: FDA Debates Creating Pilot to Evaluate Novel Excipients, Regulatory Focus 4 December 2019).
At that time, Janssen wrote that use of these excipients would go a long way towards resolving formulation challenges with certain biologics.  “Novel excipients that can stabilize proteins from adsorption at air-water and solid-water interfaces in the presence of enzymes would greatly benefit the development of biologic formulations.”
The company further added that “an excipient manufacturer’s investment in production, impurity controls, and characterization of a novel excipient would likely be spurred by the opportunity to gain FDA recognition of a novel excipient’s acceptability without tying these investments to the risk of [a new molecular entity]-specific filing.”
Proposals are being accepted until 7 December 2021.
FDA novel excipients pilot
FDA novel excipients pilot outline
Janssen comment



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