Sarepta has turned a controversial approval into a growing business
Yesterday, the FDA accepted Sarepta Therapeutics’ application for a third drug to treat Duchenne muscular dystrophy, promising to render a decision by Feb. 25. If the treatment is approved, which is widely expected, Sarepta would have a treatment option for nearly 30% of patients with DMD.
Sarepta’s current fortunes mark a stark contrast with its situation in 2016, when the approval of the company’s first medicine led to FDA infighting and widespread concern that the agency had become too lax in its approach to treatments for rare diseases. That first drug was approved for about 13% of DMD patients. Its successor, approved in December, added another 8%, and the next one would add 8% more.
Sarepta has five similar treatments in preclinical development, each promising to expand the company’s patient base. Much like Vertex Pharmaceuticals built an enviable business by developing successive medicines for cystic fibrosis, Sarepta is on a path to do the same for DMD, something nearly unimaginable four years ago.
Sarepta’s current fortunes mark a stark contrast with its situation in 2016, when the approval of the company’s first medicine led to FDA infighting and widespread concern that the agency had become too lax in its approach to treatments for rare diseases. That first drug was approved for about 13% of DMD patients. Its successor, approved in December, added another 8%, and the next one would add 8% more.
Sarepta has five similar treatments in preclinical development, each promising to expand the company’s patient base. Much like Vertex Pharmaceuticals built an enviable business by developing successive medicines for cystic fibrosis, Sarepta is on a path to do the same for DMD, something nearly unimaginable four years ago.
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