FDA Reverses Course And Approves Once-Rejected Treatment For Duchenne Muscular Dystrophy
In August, the maker of the treatment, Sarepta, said the FDA rejected the drug over the risk of infections seen in animal experiments. The new approval announcement made no mention of the prior rejection.
FDA Approves Sarepta's Once-Rejected Muscular Dystrophy Drug
The Food and Drug Administration reversed its decision on a treatment for Duchenne muscular dystrophy from Sarepta Therapeutics (SRPT), approving a previously rejected drug without explaining what the problem was in the first place. The therapy, called Vyondys 53, is approved to treat the roughly 8% of Duchenne patients whose disease results from a specific DNA error. In a clinical trial, Sarepta’s drug produced a small increase in an important muscle protein called dystrophin that is normally missing in children with Duchenne. (Garde, 12/12)
Chris Garabedian On Sarepta's Legacy And Biogen's Future At The FDA
This September marked the third anniversary of a pivotal moment in biotech. Back in 2016, Sarepta Therapeutics (SRPT) convinced the Food and Drug Administration to approve a treatment for Duchenne muscular dystrophy based on preliminary evidence from a tiny clinical trial. Critics — and there were many — argued that Sarepta hadn’t done enough to prove that its drug could actually help patients. And there were countless tweets and editorials claiming that the FDA had set a dangerous precedent by approving the company’s medicine. (Feuerstein, Garde and Robbins, 12/13)
In other pharmaceutical news —
PhRMA Sues Oregon Over Drug Pricing Laws, Calling Them 'Unconstitutional'
The trade group for the pharmaceutical industry has filed a lawsuit alleging a pair of Oregon laws is unconstitutional, the latest bid to push back against state efforts to shed more light on the rising cost of prescription medicines. One law required drug makers to notify the state when list prices rise by at least 10% or a new medicine is introduced that costs more than $670 for a month’s supply. (Silverman, 12/12)