Sarepta shares slide again as drugmaker bows to FDA pressure to pause gene therapy
The decision, announced late Monday, comes just days after the company rebuffed FDA regulators in an extremely unusual decision that alarmed investors and analysts.
Sarepta CEO Doug Ingram said the company seeks a 'productive and positive' relationship with FDA and that 'maintaining that productive working relationship required this temporary suspension.'
The Cambridge, Massachusetts-based company said it would ' temporarily pause all shipments' of its gene therapy Elevidys for muscular dystrophy at the close of business Tuesday.
It's the latest in a series of highly irregular moves that have rocked company shares for weeks and forced it to lay off hundreds of staffers.
Elevidys is the first gene therapy approved in the U.S. for Duchenne's muscular dystrophy, the fatal muscle-wasting disease that affects boys and young men, resulting in early death. The one-time treatment was initially approved for boys age 4 and younger who could still walk. Last year, FDA expanded approval to older patients who are no longer able to walk.
The therapy was already under FDA scrutiny after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. Then the company last week disclosed a third death with a different therapy: a 51-year-old patient who was enrolled in a company trial for a another form of muscular dystrophy.
FDA responded by asking the company to immediately halt all shipments of Elevidys.
Wall Street analysts said the company made the right move.
Defying the FDA would have 'irreparably damaged the company's relationship with FDA under the current leadership and administration,' TD Cowen analyst Ritu Baral told investors in a note Tuesday.
Baral estimated the pause in distribution would last three to six months.
The FDA has the authority to pull drugs from the market, but the process can take months or even years. Instead, the agency usually makes an informal request and companies almost always comply. Even in the rare cases when drugmakers haven't cooperated, the FDA has prevailed after public hearings and appeals.
___
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute's Department of Science Education and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.
Hashtags

Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
3 hours ago
- Yahoo
Madrigal (MDGL) Secures $500M to Fuel MASH Pipeline Expansion
Madrigal Pharmaceuticals (NASDAQ:MDGL) is one of the best biotech stocks to buy according to billionaire Steve Cohen. On July 22, MDGL announced a $500 million senior secured credit facility with Blue Owl Capital Inc. (NYSE:OWL), including a $350 million initial term loan and a $150 million delayed draw option through 2027. A doctor wearing a lab coat in a research laboratory, holding up a flask of a potential biotechnology therapeutic solution. The non-dilutive funding will refinance $115 million in existing debt and support strategic growth, with potential for an additional $250 million. Madrigal maintains a strong current ratio of 5.91, signaling solid liquidity. The capital will primarily advance Madrigal's pipeline for metabolic dysfunction-associated steatohepatitis (MASH), led by its FDA-approved drug Rezdiffra—the first treatment for MASH with moderate to advanced fibrosis. A Phase 3 trial is underway to evaluate Rezdiffra for compensated MASH cirrhosis. With strong launch momentum and a patent expected to extend protection through 2044, Madrigal is positioned to lead in MASH therapeutics. Madrigal Pharmaceuticals (NASDAQ:MDGL) is a biotech company that develops and commercializes novel therapies for metabolic dysfunction-associated steatohepatitis (MASH), also known as non-alcoholic steatohepatitis (NASH). While we acknowledge the potential of MDGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: and Goldman Sachs REIT Stocks: Top 12 Stock Picks. Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
4 hours ago
- Yahoo
Madrigal (MDGL) Secures $500M to Fuel MASH Pipeline Expansion
Madrigal Pharmaceuticals (NASDAQ:MDGL) is one of the best biotech stocks to buy according to billionaire Steve Cohen. On July 22, MDGL announced a $500 million senior secured credit facility with Blue Owl Capital Inc. (NYSE:OWL), including a $350 million initial term loan and a $150 million delayed draw option through 2027. A doctor wearing a lab coat in a research laboratory, holding up a flask of a potential biotechnology therapeutic solution. The non-dilutive funding will refinance $115 million in existing debt and support strategic growth, with potential for an additional $250 million. Madrigal maintains a strong current ratio of 5.91, signaling solid liquidity. The capital will primarily advance Madrigal's pipeline for metabolic dysfunction-associated steatohepatitis (MASH), led by its FDA-approved drug Rezdiffra—the first treatment for MASH with moderate to advanced fibrosis. A Phase 3 trial is underway to evaluate Rezdiffra for compensated MASH cirrhosis. With strong launch momentum and a patent expected to extend protection through 2044, Madrigal is positioned to lead in MASH therapeutics. Madrigal Pharmaceuticals (NASDAQ:MDGL) is a biotech company that develops and commercializes novel therapies for metabolic dysfunction-associated steatohepatitis (MASH), also known as non-alcoholic steatohepatitis (NASH). While we acknowledge the potential of MDGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: and Goldman Sachs REIT Stocks: Top 12 Stock Picks. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
9 hours ago
- Yahoo
Privateer Rum CEO Andrew Cabot was on overseas work trip when his wife was embroiled in Coldplay ‘kiss cam' scandal: source
Privateer Rum CEO Andrew Cabot was on an overseas business trip when his wife, Kristin Cabot, was caught canoodling at a Coldplay concert — and the couple's relatives were likewise blindsided by the scandal, a source close to the situation told The Post. The tipster said Andrew Cabot, founder of the Massachusetts-based, high-end liquor maker, had been on a work-related trip to Japan and returned to find himself and his family in the middle of a social media firestorm. [Andrew] was in Asia for a few weeks and returned on Saturday, so he was gone while all this went down,' said the source, who requested anonymity to discuss the situation. 'Poor guy.' Multiple attempts to contact Andrew Cabot and Privateer Rum went unanswered. When The Post called Privateer Rum, the company's voicemail box was full. Kristin and Andrew Cabot married sometime after her previous divorce was finalized in 2022. The source said there had been little indication of any trouble in paradise before the Coldplay concert video went viral. 'The family is now saying they have been having marriage troubles for several months and were discussing separating — which I find interesting since as of a month ago they were saying how in love they are,' the source added. The Post was first to report that Kristin Cabot, whose maiden name in Stanek, was married to Andrew Cabot and that the pair had purchased a $2.2 million mansion in Rye, New Hampshire, just five months before the scandal broke out. The Cabots were seen smiling and wearing wedding rings alongside two young kids in a Facebook post from May 2024. Andrew Cabot is a member of one of the oldest and wealthiest families in Boston. The Cabots are one of the original 'Boston Brahmin' clans that controlled New England for centuries. A bio on Privateer's website says his 'ancestor, the original Andrew Cabot (1750-1791), was a merchant, rum distiller and successful American privateer during the American Revolution.' The brand — whose high-end bottles can retail for upward of $100 each — touts its strict use of authentic ingredients and 'Made in the USA' pedigree. 'One thing is certain,' one ad advises. 'Privateer advocates transparency, honesty and purity.' Kristin Cabot's now-deleted LinkedIn account showed that she has served as an 'advisory board member' at Privateer Rum since September 2020. Andy Byron, the now-ousted CEO of New York-based software firm Astronomer, was caught canoodling on the jumbotron 'kiss cam' at Gillette Stadium with Kristin Cabot, the company's head of HR. Both scrambled to get out of sight, prompting Coldplay frontman Chris Martin to joke, 'Either they're having an affair or they're just very shy.' Astronomer placed both Kristin Cabot and Byron on leave while it investigated the incident. By last Saturday, Byron had resigned, with Astronomer co-founder Pete DeJoy taking over as the new interim CEO. So far, Kristin Cabot has yet to be fired. Solve the daily Crossword