'N of 1' ther­a­pies ad­dressed in draft FDA guid­ance

Rec­og­niz­ing the pace at which drug de­vel­op­ers are mov­ing ever fur­ther in­to in­di­vid­u­al­ized med­i­cine, the 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).

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