Updated: FDA approves Sarepta’s Duchenne muscular dystrophy gene therapy via accelerated pathway

The FDA grant­ed ac­cel­er­at­ed ap­proval to Sarep­ta Ther­a­peu­tics’ Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py for boys aged 4 and 5 in a de­ci­sion that al­lows ac­cess to the first one-time treat­ment for the de­bil­i­tat­ing mus­cle dis­ease.

The gene ther­a­py, known as SRP-9001 or de­landis­tro­gene mox­epar­vovec, will be mar­ket­ed as Ele­v­idys, Sarep­ta an­nounced Thurs­day af­ter­noon. Ele­v­idys will cost $3.2 mil­lion per pa­tient, mak­ing it the sec­ond most ex­pen­sive med­i­cine in the US af­ter CSL Behring and UniQure’s he­mo­phil­ia B gene ther­a­py Hem­genix, which costs $3.5 mil­lion per pa­tient.

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