Sareptas Golodirsen snub highlights atypical communications, heralds higher bar at FDA- analysts

Fiercebiotech | August 20, 2019

The FDA dealt a surprise rejection to Sarepta Therapeutics Monday, telling the company in a Complete Response Letter CRL that it was passing its second Duchenne muscular dystrophy DMD drug over due to risks of infusion port infection and kidney toxicity seen in animal studies. Sareptas first move will be to meet with the FDA to negotiate a black box warning or other labeling for the drug, golodirsen, to spell out the risk of kidney toxicity and infection risk, rather than to carry out more studies, wrote Evercore ISI analysts in a note Tuesday. But the implications of the rejection range beyond golodirsen. “The real driver(s) of the aytpical golodirsen CRL will continue to be a debate based on limited public disclosure of the granularity of facts, but the macro take home is clear in our minds—there is a regulatory “raising of the bar,” the analysts wrote. This could spell bad news for Sarepta’s other programs, namely its microdystrophin gene therapy that the Evercore ISI analysts consider the “major valuation driver of [Sarepta] stock.”

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Healthcare organizations are working hard to meet constituent demands for better IT services and efficiency. With huge investments in their 3-tier systems and other aging infrastructure, they are now seeking better ways to innovate and eliminate legacy complexity. This paper explains how Nutanix enables healthcare organizations to transform their static and complex IT environments into more dynamic and easily managed datacenters, bringing a cloud-like experience to IT. This paper presents several ways that healthcare organizations can achieve operational efficiencies with Nutanix.

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