FDA un­veils new draft guid­ance to help with oligonu­cleotide ther­a­peu­tics de­vel­op­ment

While oligonu­cleotides, a wide va­ri­ety of syn­thet­i­cal­ly mod­i­fied RNA or RNA/DNA hy­brids that bind to a tar­get RNA se­quence to al­ter RNA and/or pro­tein ex­pres­sion, have been win­ning ap­provals in re­cent years (e.g. No­var­tis’ cho­les­terol drug Leqvio), the reg­u­la­to­ry agency is of­fer­ing new draft guid­ance for those look­ing to fol­low a sim­i­lar path.

The non-bind­ing guid­ance, ti­tled “Clin­i­cal Phar­ma­col­o­gy Con­sid­er­a­tions for the De­vel­op­ment of Oligonu­cleotide Ther­a­peu­tics Guid­ance for In­dus­try” deals with phar­ma­co­ki­net­ic, phar­ma­co­dy­nam­ic, and safe­ty as­sess­ments re­quired as part of oligonu­cleotide ther­a­peu­tics R&D.

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