FDA Offers Advice on Nonclinical Studies for Enzyme Replacement Therapies

No single nonclinical program can be designed to fit all enzyme replacement therapies (ERT), the FDA says in a new guidance on nonclinical studies for such products.

The agency urges sponsors to use animal disease models for toxicity testing as it may allow them to detect toxicity caused by the interaction of the drug and the disease in ways that would not be observed in healthy animals.

Some toxicology assessments may not fully comply with good laboratory practice (GLP) regulations, in which case, sponsors must explain the reason for the noncompliance in the final study report. For example, toxicology data are sometimes collected in studies that may involve “unique animal-care issues and technical expertise unavailable at a GLP testing facility,” the agency says.

Because nonclinical testing programs for ERT products often need to be highly individualized, the agency urges sponsors to communicate with CDER early in the product’s development so the center can outline its expectations for the specific product and indication.