The Food and Drug Administration has finalised a guidance which will assist sponsors in designing and conducting non-clinical studies of investigational ERT products

Enzyme Replacement Therapy products are used to treat a wide range of metabolic disorders which have resulted from inherited defective genes (for example; Gaucher disease, Fabry disease or Pompe disease).

These rare, life-threatening conditions normally manifest in early life and are treated by exogenously supplying the missing or defective protein. However, the product does not exactly resemble the endogenous product due to chemical modifications for extending half-lives or targeting delivery to specific sites of the body, therefore it can lead to potential toxicities. Due to the wide variety of enzyme product types and clinical indications, a generic non-clinical program cannot be designed for use with all ERT products.

The guidance outlines recommendations for non-clinical study considerations of ERT products, however it does not apply to the development of pancreatic enzyme products. These non-clinical studies should address pharmacodynamic characterisations, safety assessments and safety parameters. The guidance also discusses various issues which may affect the design of a non-clinical programme, including selecting animal species, animal models of disease, toxicology studies, good laboratory practice and product development for clinical trials and marketing applications.

To view the full guidance, click here.