Sarep­ta dos­es first pa­tient with Duchenne gene ther­a­py fol­low­ing ac­cel­er­at­ed ap­proval

Just over a month af­ter re­ceiv­ing ac­cel­er­at­ed ap­proval from the FDA, Sarep­ta Ther­a­peu­tics has giv­en the first com­mer­cial dose of its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py to a pa­tient.

The pa­tient, a 5-year old boy named Hi­ram, was dosed at Chil­dren’s Na­tion­al Hos­pi­tal in Wash­ing­ton, DC. He re­ceived the dose one day be­fore his sixth birth­day, ac­cord­ing to the hos­pi­tal.

In June, the FDA grant­ed ac­cel­er­at­ed ap­proval to Sarep­ta for its gene ther­a­py, which Sarep­ta mar­kets as Ele­v­idys at a price tag of $3.2 mil­lion per pa­tient. That makes it the sec­ond most ex­pen­sive med­i­cine in the US af­ter CSL Behring and uniQure’s he­mo­phil­ia B gene ther­a­py Hem­genix, which is $3.5 mil­lion per pa­tient.

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