A Winning Day for Orphan Drugs!


This morning, the Food and Drug Administration announced that it granted accelerated approval to Sarepta Therapeutics’ drug Eteplirsen. This comes after months of waiting for a decision after an extremely intense Adcomm meeting. 

The decision shows the FDA is capable of retiring prehistoric data requirements and embracing the real life manifestation of a drug’s success. The boys in the Eteplirsen trial are doing well, retaining abilities and slowing the progression of a fatal disease that until today, had no treatment.

This cause is near and dear to my heart, as I have many friends with sons impacted by Duchenne. There is still plenty of work to be done, but the FDA made the right choice in accelerating approval. This sets the stage for more orphan drugs for more rare diseases.

Hats off to the Duchenne community for their hard work in presenting the data, putting the patients first and not letting a broken regulatory process discourage you.

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