'N of 1' therapies addressed in draft FDA guidance

Recognizing the pace at which drug developers are moving ever further into individualized medicine, the US Food and Drug Administration (FDA) has issued a draft guidance addressing submission processes for some hyper-specialized treatments.

The new document, which gives high-level guidance for investigational new drug (IND) submissions of individualized anti-sense oligonucleotides (ASOs), takes into consideration that these “N of 1” therapies come with “a set of challenges and considerations not seen with the typical drug intervention,” according to a statement from Patrizia Cavazzoni, MD, the acting director of FDA’s Center for Drug Evaluation and Research (CDER).