| Though Sarepta was able to push its first drug through a controversial approval three years ago, its second has met a different fate. The FDA rejected golodirsen this week—the follow-up to Exondys 51 for Duchenne muscular dystrophy—in part due to kidney toxicity in animal studies and infection risks with infusion ports. Sarepta’s stock dipped 20%, and analysts say this could be a sign of a higher bar for approval at the FDA—and potentially bad news for the company’s microdystrophin gene therapy that's currently in the works. | |
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Featured Story | | | Friday, August 16, 2019 The second of AbbVie’s highly anticipated 2019 blockbuster candidates is here. Friday, the FDA greenlighted Rinvoq, a rheumatoid arthritis med meant to succeed best-selling Humira as copycats bite into its sales. The new product should be available in the U.S. by late August, AbbVie said. |
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Top Stories Of The Week Monday, August 19, 2019 Levi Garraway is set to take up one of the biggest hot seats in biopharma when he becomes the next chief medical officer and head of global product development at Roche. Monday, August 19, 2019 Until recently, Novartis CEO Vas Narasimhan seems to have navigated through several scandals unscratched. But he could have handled the Zolgensma data manipulation crisis better, he told managers during an internal call. Monday, August 19, 2019 Sarepta pushed its first drug through approval, but its second drug is facing a different fate. The FDA rejected the New Drug Application for golodirsen, the follow-up to Exondys 51, Sarepta’s first treatment for Duchenne muscular dystrophy. Tuesday, August 20, 2019 Sarepta’s first move after its golodirsen rejection will be to negotiate a warning or other labeling for the drug rather than carry out more studies. But the implications of the rejection range beyond golodirsen. Monday, August 19, 2019 Pfizer knew things would take a turn for the worse after generics for its blockbuster Lyrica launched stateside three short weeks ago. But did the company know it would lose this much—and so fast? Monday, August 19, 2019 Alcon has announced plans to launch its daily disposable contact lens in the U.S. made from a new material. Wednesday, August 21, 2019 Pfizer is running behind in the race for a gene therapy and is shifting its production efforts into overdrive. The company will inject another $500 million into a gene therapy manufacturing plant and says it will hire another 300 workers. Thursday, August 22, 2019 Researchers at Roche and the Paul Scherrer Institute have identified a way to prevent the spread of certain cancer cells. Then they discovered that Merck already has a drug in development that may accomplish that feat. Friday, August 16, 2019 Amarin may still be waiting for the FDA to include new risk-reduction info to Vascepa's label, but in the meantime, it's getting prepared. The company is testing a pilot TV and digital ad program in select markets for the triglyceride fighter. | This week's sponsor is ExL Events. | |  | |
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