Medicine

STAT+: Pharmalittle: Sarepta wins another FDA nod; Germans balk at AstraZeneca vaccine

This post originally appeared on StatNews.

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.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED

What is it?

STAT+ is STAT’s premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What’s included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.

This post originally appeared on StatNews.

Orange in Life

Subscribe to Our Newsletter