STAT+: Pharmalittle: Sarepta wins another FDA nod; Germans balk at AstraZeneca vaccine
Good morning, one and all. Damian Garde here, filling in for Ed Silverman in the waning hours of a normal workweek made to feel that much longer by the truncated one that preceded it. The weather is slowly tilting toward the pleasant in at least this part of the world, an invitation to spend some time outdoors for the weekend hours not devoted to reloading vaccine appointment webpages. Until then, here as always are some tidbits to get your day started.
Sarepta Therapeutics won FDA approval for its third drug targeting Duchenne muscular dystrophy, STAT reports. The treatment, called Amondys 45, is for the roughly 8% of Duchenne patients whose disease is tied to a specific genetic mutation. Sarepta’s three approved medicines cover about 30% of patients with the disease. Amondys 45, like Sarepta’s other drugs, was based on its ability to produce a tiny amount of the muscle protein dystrophin, even though there is no established proof that more dystrophin improves muscle function or slows the progression of the disease.
Continue to STAT+ to read the full story…
