The FDA today released draft guidance designed to make it easier for researchers to seek approvals for drugs designed for individuals with severe genetic diseases.
The draft guidance focuses on investigational new drug submissions for antisense oligonucleotide (ASO) products because these are the most common drugs being developed for severely debilitating or life-threatening genetic diseases. ASOs target messenger RNA and may help people for whom there is no adequate available therapy and who need prompt medical intervention because of rapid disease progression.
The draft guidance also targets academic investigators who may be less familiar with FDA regulations, policies and practices, and less experienced than biopharmaceutical or pharmaceutical companies in working with the agency. It includes information on:
The approach to obtaining feedback from the FDA. The expectations and process for making regulatory submissions to the agency. Recommendations abou…