Environmental Risk Assessment for a New Drug Application of a Novel Active Pharmaceutical Ingredient

OUR CHALLENGE

Our client needed ecotoxicological expertise on a time-sensitive inquiry by U.S. Food and Drug Administration (FDA) on a new treatment for a rare and devastating childhood disease. The agency requested supporting information for the active pharmaceutical ingredient (API) to qualify for categorical exclusion from an environmental risk assessment under a new drug application.

OUR APPROACH

We provided comprehensive scientific justification that included bridging ecotoxicological data from other APIs with a comprehensive read-across justification, in silico modeling of environmental fate and decomposition products, and discussion of kinetics and pharmacodynamics. We also employed environmental exposure models to derive predicted environmental concentrations. Integral presented the collective evidence to FDA in an agency-hosted video conference call and subsequently provided the client with a report for submission to FDA.

OUR IMPACT

FDA accepted Integral’s multiple lines of evidence without the need for new experimental data. A categorical exclusion from an environmental risk assessment was granted and the client received FDA approval of the new API to treat Duchenne muscular dystrophy.