{"id":41740,"date":"2025-07-19T11:35:49","date_gmt":"2025-07-19T11:35:49","guid":{"rendered":"https:\/\/quickassetsmarket.com\/index.php\/2025\/07\/19\/sarepta-shares-fall-37-as-fda-questions-future-of-key-gene-therapy\/"},"modified":"2025-07-19T11:35:49","modified_gmt":"2025-07-19T11:35:49","slug":"sarepta-shares-fall-37-as-fda-questions-future-of-key-gene-therapy","status":"publish","type":"post","link":"https:\/\/quickassetsmarket.com\/index.php\/2025\/07\/19\/sarepta-shares-fall-37-as-fda-questions-future-of-key-gene-therapy\/","title":{"rendered":"Sarepta shares fall 37% as FDA questions future of key gene therapy"},"content":{"rendered":"
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Shares of Sarepta Therapeutics fell close to 37% on Friday after reports emerged that the US Food and Drug Administration is considering whether to halt the distribution of Elevidys, the company\u2019s flagship gene therapy for Duchenne Muscular Dystrophy (DMD). <\/p>\n

The move comes amid an ongoing investigation into multiple patient deaths tied to Sarepta\u2019s gene therapy programs.<\/p>\n

A source familiar with the matter told CNBC that the FDA is preparing to request Sarepta voluntarily suspend shipments of Elevidys, though the agency has yet to formally contact the company. <\/p>\n

Sarepta said it had not received any direct communication from the FDA as of Friday.<\/p>\n

Separately, FDA Commissioner Marty Makary confirmed to Bloomberg News that the agency is reviewing whether Elevidys should remain on the market, raising fears that Sarepta\u2019s primary commercial product could be withdrawn entirely.<\/p>\n

Deaths reignite long-standing safety concerns<\/h2>\n

The FDA\u2019s concerns follow the deaths of two teenage boys from liver failure earlier this year after receiving Elevidys. <\/p>\n

A third patient, enrolled in a Phase 1 trial for a different Sarepta gene therapy, also died recently. <\/p>\n

While the therapies differ in their targets, both use the same delivery mechanism, fueling broader safety concerns.<\/p>\n

Elevidys has been controversial from the outset. <\/p>\n

Initially granted conditional approval in 2023 for DMD patients aged 4 to 5 who could still walk, its approval was expanded in 2024 to include all ambulatory patients aged 4 and up, and conditionally for non-ambulatory patients. <\/p>\n

The latter decision drew criticism, as clinical data showed weaker evidence of efficacy in this group.<\/p>\n

Further concerns arose when Elevidys failed to meet its primary goal in a Phase 3 trial. <\/p>\n

Nevertheless, former FDA official Peter Marks sided with Sarepta, citing secondary benefits in the trial to justify expanded approval, a move that has since come under renewed scrutiny.<\/p>\n

Baird downgrades Sarepta stock; analysts warn of dire consequences if Elevidys approval revoked <\/h2>\n

The regulatory probe has dealt a blow to Sarepta, which relies heavily on Elevidys for its revenue. <\/p>\n

The therapy accounted for more than half of Sarepta\u2019s total net product revenue. <\/p>\n

With the company\u2019s shares now down more than 87% year-to-date, investors are bracing for the possibility that the FDA may revoke approval altogether.<\/p>\n

Analyst Kostas Biliouris of BMO Capital Markets noted that while other gene therapies, such as Novartis\u2019s Zolgensma, have also faced safety issues, the clear benefit of Zolgensma justifies the risk.<\/p>\n

\u201cThat\u2019s why deaths here matter so much versus Zolgensma, for example,\u201d Biliouris said.<\/p>\n

The company has stopped shipping Elevidys to non-ambulatory patients while it assesses alternative delivery methods. <\/p>\n

Still, Sarepta executives insist the drug could bring in $500 million annually even if limited to treating ambulatory patients.<\/p>\n

The company\u2019s future now hinges on the FDA\u2019s decision. \u201cIf the FDA pulls Elevidys from the market,\u201d Biliouris warned, \u201cSarepta is done.\u201d<\/p>\n

“Although the development of SRP-9004 has been discontinued, we believe the death could lead to greater scrutiny of SRP-9003’s safety profile ahead of its BLA submission and could affect commercial interest, if approved,” brokerage William Blair said. <\/p>\n

Baird downgraded Sarepta Therapeutics to Neutral from Outperform, with a $15 Price Target.<\/p>\n

The post Sarepta shares fall 37% as FDA questions future of key gene therapy<\/a> appeared first on Invezz<\/a><\/p>\n

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Shares of Sarepta Therapeutics fell close to 37% on Friday after reports emerged that the US Food and Drug Administration is considering whether to halt the distribution of Elevidys, the company\u2019s flagship gene therapy for Duchenne Muscular Dystrophy (DMD). The move comes amid an ongoing investigation into multiple patient deaths tied to Sarepta\u2019s gene therapy <\/p>\n","protected":false},"author":1,"featured_media":41741,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[5],"tags":[],"class_list":{"0":"post-41740","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-investing"},"_links":{"self":[{"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/posts\/41740","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/comments?post=41740"}],"version-history":[{"count":0,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/posts\/41740\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/media\/41741"}],"wp:attachment":[{"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/media?parent=41740"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/categories?post=41740"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/quickassetsmarket.com\/index.php\/wp-json\/wp\/v2\/tags?post=41740"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}