Inside Precision Medicine June 21, 2024
Sarepta is shining, as its Duchenne Muscular Dystrophy (DMD) gene therapy Elevidys has been granted the much wider label the biotech was aiming for—making it available to four-year-olds and older. This news comes just about a week after Pfizer, which was fast on Sarepta’s heels with their own gene therapy for DMD, suffered a key Phase III trial failure.
Sales of Elevidys were $200.4 million in 2023, significantly exceeding estimates. Helping net the company over $1.145 billion last year: Sarepta also has several phosphorodiamidate morpholino oligomer (PMO) chemistry-based exon-skipping marketed therapies for DMD as well.
Sarepta almost missed a beat though when it reported, in Oct. 2023, that its pivotal Phase III of Elevidys failed on the primary...
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