A for­mer fail­ure in PhII, the go-go FDA says it’s ready to con­sid­er an ac­cel­er­at­ed short­cut for Ul­tragenyx's UX007

More than a year af­ter Ul­tragenyx $RARE an­nounced the fail­ure of a Phase II study for its seizure med­ica­tion UX007, the rare dis­ease spe­cial­ist says the FDA has now of­fered a thumbs-up for its will­ing­ness to con­sid­er an ac­cel­er­at­ed ap­proval for a dif­fer­ent in­di­ca­tion.

The biotech is pur­su­ing an OK for long-chain fat­ty acid ox­i­da­tion dis­or­ders af­ter piec­ing to­geth­er da­ta that spot­light­ed a drop in ma­jor clin­i­cal events for LC-FAOD.

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