The US FDA verdict on Sarepta's Elevidys, a gene therapy delivering microdystrophin to patients with DMD, proved to be the most controversial decision of 2Q23, according to our drug pipeline Data Page
The US Food and Drug Administration verdict on Sarepta’s Elevidys, a gene therapy delivering microdystrophin to patients with DMD, proved to be the most controversial decision of the quarter. The company teased positive efficacy from the data for patients aged 4–5 but not for older peers, with Sarepta’s fervent promise of data by the fall from a randomized trial in that narrow patient band clinching an accelerated approval.
Between the extremes of complexity falls Krystal Biotech’s Vyjuvek, a redosable gene therapy for the severe skin disorder dystrophic epidermolysis bullosa, caused by inherited deficiencies in collagen VII: Vyjuvek is the first approved topically applied gene therapy and the first based on herpes simplex virus-1 .
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