Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarep­ta may be run­ning in­to some trou­ble with its next-gen gene ther­a­py ap­proach to Duchenne mus­cu­lar dy­s­tro­phy. But when it comes to an­ti­sense oligonu­cleotides, the well-trod­den reg­u­la­to­ry path is still lead­ing straight to an ac­cel­er­at­ed ap­proval for casimersen, now chris­tened Amondys 45.

We just have to wait un­til 2024 to find out if it works.

Amondys 45’s ap­proval was un­cer­e­mo­ni­ous, com­pared to its two old­er sib­lings. There was no con­tro­ver­sy with­in the FDA over ap­prov­ing a drug based on a bio­mark­er rather than clin­i­cal ben­e­fit, set­ting up a pow­er­ful prece­dent that still haunts act­ing FDA com­mis­sion­er Janet Wood­cock as biotech in­sid­ers weighed her po­ten­tial per­ma­nent ap­point­ment; no dra­ma like the FDA is­su­ing a stun­ning re­jec­tion on­ly to re­verse its de­ci­sion and hand out an OK four months lat­er, which got more com­pli­cat­ed af­ter the scathing com­plete re­sponse let­ter was pub­lished; no anx­ious tea leaf read­ing or heat­ed ar­gu­ments from drug de­vel­op­ers and pa­tient ad­vo­cates who were tired of hav­ing cor­ti­cos­teroids as their loved ones’ on­ly (some­times ex­pen­sive) op­tion.

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