CDER (Center for Drug Evaluation and Research) recently launched a new voluntary Novel Excipient Review Pilot Program, intended to allow excipient manufacturers to obtain FDA review of certain ‘novel excipients’ prior to their use in medicinal products. This pilot is geared at fostering development of excipients that may be useful in scenarios in which manufacturers and originators have difficulties in using existing excipients.

This pilot will initially 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.

For excipient manufacturers, the pilot consists of two stages: The first stage is to provide a high-level overview of the ‘novel excipient’, with stage two providing a full data set including toxicology and quality data.

What is a ‘novel excipient’?

Excipients are inactive ingredients that agencies review as a component of a finished drug. This could be in a new drug application but also in a generic drug application. These inactive ingredients can work to stabilise medications and help the body absorb the active ingredients. Other areas of use are for flavouring, colouring or coating of the medicinal product. ‘Novel excipients’ are any excipients that are not fully supported by existing safety data yet.

Why this pilot then?

Excipients can certainly help with delivering drugs to where they are needed in the body. They can also play a part in helping patients to tolerate medication, but sometimes this is associated with risks. Triggering allergies for example is one area of concern. Hence regulators evaluate excipients for safety and stability as early as possible, so as not to negatively impact any medicinal product delivery to the patients. With this pilot, the FDA opens a pathway to evaluate ‘novel excipients’ that could provide important public health benefits. Submissions are voluntary, allowing excipient manufacturers to obtain early review of certain ‘novel excipients’ prior to their use.

How are ‘novel excipients’ selected?

The FDA will accept a limited number of ‘novel excipients’ for this pilot. Selection preference will be given to proposals demonstrating

• a potential public health benefit – one example being excipients promoting development of new therapies for serious diseases.
• potential to improve characteristics that may lead to new drug development
• likelihood of timely delivery of complete package ready for assessment.

In summary

The ‘novel excipient’ pilot will work to promote development and use of new excipients, where using existing excipients is challenging or not possible. Its success will be measured with the review and identification of ‘novel excipients’ that improve drug development including new medicinal products, to be introduced to the patients for an unmet medical need.

Published by Olaf Schoepke

Passionate about sharing ideas with regulatory professionals in the human and veterinary medicinal product space.

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