Taysha stops de­vel­op­ing a gene ther­a­py amid de­bate over FDA’s stance on ul­tra-rare tri­als

Taysha Gene Ther­a­pies dis­con­tin­ued de­vel­op­ment of a neu­rode­gen­er­a­tive ther­a­py af­ter the FDA again in­sist­ed on a po­ten­tial­ly im­pos­si­ble study, the com­pa­ny said Tues­day.

It’s yet an­oth­er re­minder of how dif­fi­cult it is to bring med­i­cines tar­get­ing ul­tra-rare con­di­tions to mar­ket.

The Dal­las-based com­pa­ny was seek­ing ap­proval of a ther­a­py aimed at gi­ant ax­on­al neu­ropa­thy (GAN), a con­di­tion that ren­ders pa­tients quad­ri­pleg­ic and is fol­lowed by ear­ly death. Taysha said its plans were dashed by the FDA re­quir­ing a ran­dom­ized, place­bo-con­trolled tri­al.

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