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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