Oxford spinout raises $113M in bid to beat Sarepta's Exondys 51 in DMD. But will we ever know if any of the drugs work?
It’s been five years since the FDA decided to approve Sarepta’s Duchenne muscular dystrophy drug Exondys 51 on a sliver of biological evidence, and still there are little data to show the drug can actually slow patients’ decline.
That paucity, though, hasn’t scared off investors from investing in companies trying to treat the disease with the same approach. On the contrary, it has given such companies a clear path to gain approval after minimal testing, while the $400-plus million in sales Sarepta has raked in from Exondys 51 has shown just how large a market there could be for any company that can show more biological effect than the large biotech.
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