USA – ‘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developers are moving ever further into individualized medicine, the US Food and Drug Administration (FDA) has issued a draft guidance addressing submission processes for some hyper-specialized treatments.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).
Cavazzoni noted that many of the rare diseases treated by tailored ASOs are rapidly progressive, so time is of the essence if the affected individual is to be helped by the treatment. Also, much ASO research and treatment is carried out in academic settings by investigators who may have less FDA experience than traditional drug developers…