Surprise Turnaround: Golodirsen is Approved

The FDA approved golodirsen (Vyondys 53, Sarepta Therapeutics) on 12/12/2019 for the treatment of Duchenne muscular dystrophy amenable to exon 53 skipping. This is a reversal of the August 2019 decision, where the FDA rejected golodirsen due to the risk of infection with intravenous infusion ports and renal toxicity seen in pre-clinical studies. 

Sarepta plans to price  golodirsen similar to eteplirsen. The average annual patient cost for eteplirsen is $300,000, but the cost can exceed $1 million because the drug is dosed by weight. A review by ICER of treatments for Duchenne’s found insufficient data that a slight increase in levels of dystrophin with eteplirsen and golodirsen provides a clinically significant effect. Due to the lack of evidence supporting a clinical benefit with the drugs, ICER did not estimate the pharmacoeconomic impact of either drug. 

Sarepta has shown that golodirsen will increase dystrophin levels, but has not completed studies to demonstrate an improvement in muscle function. The FDA is requiring Sarepta to complete a clinical efficacy study, so the company is recruiting at least 45 boys for the Phase III ESSENCE trial that will compare golodirsen to placebo on the impact of a 6-minute walking test. The trial is projected to be completed in 2024.